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DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have accessTACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applicationsTERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TINA DUONG - TREAT-NMD Tina Duong is a research physical therapist at Stanford University with over 18 years of experience in the clinic and neuromuscular research. Over the past 13 years, she has been instrumental in optimizing clinical endpoints for study design and assessments in both human and animal studies through her work in neuromuscular clinic, theCINRG network
2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have accessTACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applicationsTERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TINA DUONG - TREAT-NMD Tina Duong is a research physical therapist at Stanford University with over 18 years of experience in the clinic and neuromuscular research. Over the past 13 years, she has been instrumental in optimizing clinical endpoints for study design and assessments in both human and animal studies through her work in neuromuscular clinic, theCINRG network
TREAT-NMD PUBLICATIONS TREAT-NMD Publications. Please find below a list of TREAT-NMD related publications. If you would like to list a publication on our website please contact info@treat-nmd.org. If you would like to submit a global registry publication to the website, please click here. DMDPublications.
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials. DIFFERENT TYPES OF REGISTRIES Different types of registries. Linking up the national registries. List of registries by disease. Congenital muscular dystrophies. Congenital Muscle Disease International Registry. Global Registry for COL6-related dystrophies. Congenital myasthenic syndromes. Charcot Marie Tooth Disease. Myotonic dystrophy. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weTACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications CURE CMD 2022 SCIENTIFIC & FAMILY CONFERENCE More details to follow. TREAT-NMD. Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world DUCHENNE MUSCULAR DYSTROPHY (DMD) CORE DATASET This page contains information about the core dataset for TREAT-NMD registries which are collecting data on individuals with DMD. This dataset was expanded in February 2021 for clinically reported registries and in May 2021 for patient reported registries in order to better inform on the natural history of DMD, and provide data to support post-marketing surveillance (safety and effectiveness DIAGNOSTIC TESTING AND CARE OF NEW SMA PATIENTS Diagnostic testing anD care of new sMa patients II. Diagnostic procedures The stepwise algorithm of the diagnostic procedure is summarized in Figure 1.A GUIDE TO THE 2017
8 A Guide to the 2017 Internationa tandard of Care for A To make the updated standards of care more accessible, TREAT-NMD worked with a number of international patient advocacy and support groups to developthis guide.
CARE AND TRIAL SITE REGISTRY Care and Trial Site Registry – CTSR. A Powerful Tool for Clinical Research in Rare Diseases. Kirsten König and Jan Kirschner. Dept. of Neuropaediatrics and Muscle Disorders TREAT-NMD PUBLICATIONS TREAT-NMD Publications. Please find below a list of TREAT-NMD related publications. If you would like to list a publication on our website please contact info@treat-nmd.org. If you would like to submit a global registry publication to the website, please click here. DMDPublications.
2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.GUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have access ASSOCIATION FRANÇAISE CONTRE LES MYOPATHIES Association Française contre les Myopathies – AFM. Created in 1958 and managed by a group of patients and their families, AFM has become a strong organisation combining the militant energy of its founding members, the professionalization of its volunteer managers and theANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. TREAT-NMD PUBLICATIONS TREAT-NMD Publications. Please find below a list of TREAT-NMD related publications. If you would like to list a publication on our website please contact info@treat-nmd.org. If you would like to submit a global registry publication to the website, please click here. DMDPublications.
2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.GUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have access ASSOCIATION FRANÇAISE CONTRE LES MYOPATHIES Association Française contre les Myopathies – AFM. Created in 1958 and managed by a group of patients and their families, AFM has become a strong organisation combining the militant energy of its founding members, the professionalization of its volunteer managers and theANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ASSOCIATION FRANÇAISE CONTRE LES MYOPATHIES Association Française contre les Myopathies – AFM. Created in 1958 and managed by a group of patients and their families, AFM has become a strong organisation combining the militant energy of its founding members, the professionalization of its volunteer managers and the ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
ANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field.DONOVAN LOTT
TREAT-NMD. Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world DIAGNOSTIC TESTING AND CARE OF NEW SMA PATIENTS Diagnostic testing anD care of new sMa patients II. Diagnostic procedures The stepwise algorithm of the diagnostic procedure is summarized in Figure 1. TREAT-NMD PUBLICATIONS TREAT-NMD Publications. Please find below a list of TREAT-NMD related publications. If you would like to list a publication on our website please contact info@treat-nmd.org. If you would like to submit a global registry publication to the website, please click here. DMDPublications.
2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, we DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to TREAT-NMD NEWSLETTER TREAT-NMD Services Limited are looking for a new Chief Executive Officer. The ideal person will have previous operational management experience, a proven track record for effective team management and results-driven leadership, preferably from working within an small and medium sized enterprises, Charitable/3rd Sector organization or HealthCare organization.
CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.GUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have accessANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. TREAT-NMD PUBLICATIONS TREAT-NMD Publications. Please find below a list of TREAT-NMD related publications. If you would like to list a publication on our website please contact info@treat-nmd.org. If you would like to submit a global registry publication to the website, please click here. DMDPublications.
2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, we DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to TREAT-NMD NEWSLETTER TREAT-NMD Services Limited are looking for a new Chief Executive Officer. The ideal person will have previous operational management experience, a proven track record for effective team management and results-driven leadership, preferably from working within an small and medium sized enterprises, Charitable/3rd Sector organization or HealthCare organization.
CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.GUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have accessANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field.ABOUT TREAT-NMD
About TREAT-NMD. Recent years have seen rapid developments in the neuromuscular field and a corresponding surge in interest from the pharmaceutical industry. Promising preclinical results raise the potential for new therapies in the near future. Yet translational research towards therapy development for neuromuscular diseases hasfaced a number
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
TREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications DUCHENNE MUSCULAR DYSTROPHY (DMD) CORE DATASET This page contains information about the core dataset for TREAT-NMD registries which are collecting data on individuals with DMD. This dataset was expanded in February 2021 for clinically reported registries and in May 2021 for patient reported registries in order to better inform on the natural history of DMD, and provide data to support post-marketing surveillance (safety and effectivenessDONOVAN LOTT
TREAT-NMD. Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
ASSOCIATION FRANÇAISE CONTRE LES MYOPATHIES Association Française contre les Myopathies – AFM. Created in 1958 and managed by a group of patients and their families, AFM has become a strong organisation combining the militant energy of its founding members, the professionalization of its volunteer managers and the TREAT-NMD.ORGWEB VIEW 2.0099999999999998. 2.02. 2.0299999999999998. 2.04. 2.0499999999999998. 2.06. 2.0699999999999998. 2.13. 2.14. 14.06. 5.04. 5.05. 6.08. 6.09. 9.0299999999999994. 9 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have accessTACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applicationsTERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
A GUIDE TO THE 2017
8 A Guide to the 2017 Internationa tandard of Care for A To make the updated standards of care more accessible, TREAT-NMD worked with a number of international patient advocacy and support groups to developthis guide.
2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to CURRENT TRIALS IN LGMD Current trials in LGMD. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trialsthat are
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGUIDE FOR FAMILIES
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy, TREAT-NMD and the World Duchenne Organisation have used updated recommendations, to work together again to produce a 2018 current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne.. To ensure as many people as possible have accessTACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applicationsTERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
A GUIDE TO THE 2017
8 A Guide to the 2017 Internationa tandard of Care for A To make the updated standards of care more accessible, TREAT-NMD worked with a number of international patient advocacy and support groups to developthis guide.
DIFFERENT TYPES OF REGISTRIES Different types of registries. Linking up the national registries. List of registries by disease. Congenital muscular dystrophies. Congenital Muscle Disease International Registry. Global Registry for COL6-related dystrophies. Congenital myasthenic syndromes. Charcot Marie Tooth Disease. Myotonic dystrophy.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications DUCHENNE MUSCULAR DYSTROPHY (DMD) CORE DATASET This page contains information about the core dataset for TREAT-NMD registries which are collecting data on individuals with DMD. This dataset was expanded in February 2021 for clinically reported registries and in May 2021 for patient reported registries in order to better inform on the natural history of DMD, and provide data to support post-marketing surveillance (safety and effectivenessDONOVAN LOTT
TREAT-NMD. Advancing diagnosis, care and treatment for people with neuromuscular diseases around the worldPETRA KAUFMANN
Petra Kaufmann, M.D., M.Sc., is Director of the Division of Clinical Innovation at the National Center for Advancing Translational Science (NCATS). In this capacity, she oversees the Clinical And Translational Science Award (CTSA) program, which supports a national consortium of medical research institutions working to transform the way biomedical research is conducted. TINA DUONG - TREAT-NMD Tina Duong is a research physical therapist at Stanford University with over 18 years of experience in the clinic and neuromuscular research. Over the past 13 years, she has been instrumental in optimizing clinical endpoints for study design and assessments in both human and animal studies through her work in neuromuscular clinic, theCINRG network
ICHIZO NISHINO
Ichizo Nishino. Director, Department of Neuromuscular Research. Japan. After having 5 years of clinical neurology training, Dr. Nishino started muscle disease research from 1994 under the tutelage of Dr. Ikuya Nonaka, who remained a steadfast source of inspiration. Later, his postdoctoral research for two years at Columbia Universitysolidified
TREAT-NMD.ORGWEB VIEW 2.0099999999999998. 2.02. 2.0299999999999998. 2.04. 2.0499999999999998. 2.06. 2.0699999999999998. 2.13. 2.14. 14.06. 5.04. 5.05. 6.08. 6.09. 9.0299999999999994. 9ALBERTO DUBROVSKY
Prof. Alberto Dubrovsky is Professor of Neurology at the University of Buenos Aires, Argentina, and Full Professor of Neurosciences at the Favaloro University in Buenos Aires. He is Director of the Department of Neurology of the Neurosciences Institute at Fundacion Favaloro, where he also leads the Neuromuscular Disease Unit. TREAT-NMD LGMD CORE DATASET Introduction Scope of this specification This dataset specification aims to leave registries as much flexibility as possible for their individual consider- 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied toTREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied toTREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
TREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. TREAT-NMD NEWSLETTER TREAT-NMD Services Limited are looking for a new Chief Executive Officer. The ideal person will have previous operational management experience, a proven track record for effective team management and results-driven leadership, preferably from working within an small and medium sized enterprises, Charitable/3rd Sector organization or HealthCare organization.
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials. INFORMATION FOR PATIENTS CONSIDERING REGISTERING WITH A First, by using the link at the end of this page, check if there is a registry for your condition in your country. If you agree to register in your own national registry, you should read the patient information and sign the consent form associated with your own national registry. TREAT-NMD.ORGWEB VIEW 2.0099999999999998. 2.02. 2.0299999999999998. 2.04. 2.0499999999999998. 2.06. 2.0699999999999998. 2.13. 2.14. 14.06. 5.04. 5.05. 6.08. 6.09. 9.0299999999999994. 9 TINA DUONG - TREAT-NMD Tina Duong is a research physical therapist at Stanford University with over 18 years of experience in the clinic and neuromuscular research. Over the past 13 years, she has been instrumental in optimizing clinical endpoints for study design and assessments in both human and animal studies through her work in neuromuscular clinic, theCINRG network
PETRA KAUFMANN
Petra Kaufmann, M.D., M.Sc., is Director of the Division of Clinical Innovation at the National Center for Advancing Translational Science (NCATS). In this capacity, she oversees the Clinical And Translational Science Award (CTSA) program, which supports a national consortium of medical research institutions working to transform the way biomedical research is conducted. TREAT-NMD LGMD CORE DATASET Introduction Scope of this specification This dataset specification aims to leave registries as much flexibility as possible for their individual consider- 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied toTREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied toTREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
TREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. TREAT-NMD NEWSLETTER TREAT-NMD Services Limited are looking for a new Chief Executive Officer. The ideal person will have previous operational management experience, a proven track record for effective team management and results-driven leadership, preferably from working within an small and medium sized enterprises, Charitable/3rd Sector organization or HealthCare organization.
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials. INFORMATION FOR PATIENTS CONSIDERING REGISTERING WITH A First, by using the link at the end of this page, check if there is a registry for your condition in your country. If you agree to register in your own national registry, you should read the patient information and sign the consent form associated with your own national registry. TREAT-NMD.ORGWEB VIEW 2.0099999999999998. 2.02. 2.0299999999999998. 2.04. 2.0499999999999998. 2.06. 2.0699999999999998. 2.13. 2.14. 14.06. 5.04. 5.05. 6.08. 6.09. 9.0299999999999994. 9 TINA DUONG - TREAT-NMD Tina Duong is a research physical therapist at Stanford University with over 18 years of experience in the clinic and neuromuscular research. Over the past 13 years, she has been instrumental in optimizing clinical endpoints for study design and assessments in both human and animal studies through her work in neuromuscular clinic, theCINRG network
PETRA KAUFMANN
Petra Kaufmann, M.D., M.Sc., is Director of the Division of Clinical Innovation at the National Center for Advancing Translational Science (NCATS). In this capacity, she oversees the Clinical And Translational Science Award (CTSA) program, which supports a national consortium of medical research institutions working to transform the way biomedical research is conducted. TREAT-NMD LGMD CORE DATASET Introduction Scope of this specification This dataset specification aims to leave registries as much flexibility as possible for their individual consider- 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied toTREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. 2021 CMD SCIENTIFIC AND FAMILY CONFERENCE 2021 CMD Scientific and Family Conference. 21 – 22 May 2021. This year’s conference will be hosted 100% online. Registration will be required for one virtual “connection” – meaning that if you will be attending the conference with family, friends or colleagues at the same location, you only need to GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
DMD RESEARCH OVERVIEW DMD Research overview. The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied toTREAT-NMD MEMBERS
The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. 19TH ASIAN-OCEANIAN MYOLOGY CENTER MEETING AOMC2021 will be hybrid (virtual plus on-site) and will take place on 10-11 June, 2021 at BEXCO, Busan. Although we were very much looking forward to welcoming all of you to Busan in 2021, we had to decided to switch AOMC2021 to a hybrid format because of the continuing COVID19 pandemic. Unfortunately, weGNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials.TACT : OVERVIEW
TACT : Overview. To date the TACT has held 20 review meetings across Europe and the US, and has reviewed a total of 46 program applications from both academic investigators and industry in the following areas: DMD, BMD, SMA, IBM, CMD, NMD, MNGIE and XLMTM. Of the 46 compounds reviewed 29 were from industry and 17 were from academic applications ULRIKE SCHARA-SCHMIDT Ulrike Schara-Schmidt. Prof. Dr. med. Germany. Ulrike Schara-Schmidt is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. Ulrike has focussed her clinical and research activities on neuromuscular disorders, especially in infants, children andadolescents.
TERENCE PARTRIDGE
Terence Partridge. Professor of Systematic Biology. United States of America. I obtained a B.Sc. in Zoology in 1962 and a Ph.D. on cell behaviour in 1970. After a year in the Musée Nationale D’Histoire Naturelle in Paris the working on rodent models of malaria and a year working as a Chemical Pathology Technician at St Mary’s HospitalANNAMARIA DE LUCA
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. GLOBAL DMD REGISTRY STATISTICS Global DMD Registry statistics. The TREAT-NMD Global DMD Registry was established in 2007 as a way of harmonizing the national DMD registries using a common dataset. As a result, information can be shared and compared between the different national registries by linking into a centralized database. A systematic review of thenational DMD
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The benefits of Organisational membership are: Organisational members will appear on the TREAT-NMD website to show they are affiliated with TREAT-NMD. 20% discount to the Journal of Neuromuscular Diseases individual yearly subscription and 10% discount to Open Access fee for papers being submitted. Section 2 - Individual Membership. TREAT-NMD NEWSLETTER TREAT-NMD Services Limited are looking for a new Chief Executive Officer. The ideal person will have previous operational management experience, a proven track record for effective team management and results-driven leadership, preferably from working within an small and medium sized enterprises, Charitable/3rd Sector organization or HealthCare organization.
THE DIAGNOSIS & MANAGEMENT OF DMD In 2010, a consensus document ‘The Diagnosis and management of Duchenne muscular dystrophy’ was published as two editions in the Lancet Neurology in January and February 2010. Part 1: Lancet Neurol. 2010 Jan; 9 (1):77-93. Click here to download the entire article(parts 1 and 2
GNE MYOPATHY
Current Trials in GNE myopathy. TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases.The resources and expertise available through the network can make it much easier for companies to carry out their trials. INFORMATION FOR PATIENTS CONSIDERING REGISTERING WITH A First, by using the link at the end of this page, check if there is a registry for your condition in your country. If you agree to register in your own national registry, you should read the patient information and sign the consent form associated with your own national registry. TREAT-NMD.ORGWEB VIEW 2.0099999999999998. 2.02. 2.0299999999999998. 2.04. 2.0499999999999998. 2.06. 2.0699999999999998. 2.13. 2.14. 14.06. 5.04. 5.05. 6.08. 6.09. 9.0299999999999994. 9 TINA DUONG - TREAT-NMD Tina Duong is a research physical therapist at Stanford University with over 18 years of experience in the clinic and neuromuscular research. Over the past 13 years, she has been instrumental in optimizing clinical endpoints for study design and assessments in both human and animal studies through her work in neuromuscular clinic, theCINRG network
PETRA KAUFMANN
Petra Kaufmann, M.D., M.Sc., is Director of the Division of Clinical Innovation at the National Center for Advancing Translational Science (NCATS). In this capacity, she oversees the Clinical And Translational Science Award (CTSA) program, which supports a national consortium of medical research institutions working to transform the way biomedical research is conducted. TREAT-NMD LGMD CORE DATASET Introduction Scope of this specification This dataset specification aims to leave registries as much flexibility as possible for their individual consider- * Skip to primary navigation* Skip to content
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ADVANCING DIAGNOSIS, CARE AND TREATMENT FOR THOSE LIVING WITH NEUROMUSCULAR DISEASES AROUND THE WORLD... STATEMENT ON COVID-19 THE COVID-19 OUTBREAK IS INCREASINGLY IMPACTING COUNTRIES WORLDWIDE, WITH MANY UNDERTAKING RESTRICTIVE MEASURES ON THEIR CITIZENS TO CONTAIN THE SPREAD OF THE VIRUS, INCLUDING RECOMMENDATIONS THAT ALL THOSE WITH A RARE DISEASE SHOULD SELF-ISOLATE FOR 12 WEEKS. AS NEUROMUSCULAR CONDITIONS ARE CONSIDERED RARE DISEASES, TREAT-NMD HIGHLY RECOMMENDS FOLLOWING THE ADVICE OF YOUR NATIONAL AUTHORITY, AND THE WORLD HEALTH ORGANISATION. THE
LATEST UPDATES FROM THE ASSOCIATION OF BRITISH NEUROLOGISTS CAN BE FOUND HERE , AND THE LATEST PRESS RELEASE FROM EURORDIS RARE DISEASES EUROPE CAN BE FOUND HERE.
TREAT-NMD ALSO RECOMMENDS THAT PATIENTS, CARERS AND HEALTHCARE PROFESSIONALS FOLLOW THE GENERAL ADVICE ABOUT COVID-19 AND THE EFFECT ON PEOPLE WITH NEUROMUSCULAR DISEASES PUBLISHED BY THE WORLD MUSCLESOCIETY . WE WOULD
ALSO LIKE TO DRAW ATTENTION TO RECORDINGS OF THE WORLD DUCHENNE ORGANISATION’S WEEKLY ONLINE WEBINARSWHICH CONTAIN
USEFUL TIPS, ADVICE AND RECOMMENDATIONS FOR THE DUCHENNE COMMUNITY. PLEASE NOTE, ANY SPECIFIC INDIVIDUAL QUESTIONS OR PROBLEMS SHOULD BE DISCUSSED WITH YOUR RELEVANT HEALTHCARE PROFESSIONALS. THE TREAT-NMD NETWORK CONTINUES TO BE OPERATIONAL DURING THE COVID-19 OUTBREAK. YOU CAN KEEP UP-TO-DATE ON OUR ACTIVITIES THROUGH OUR WEBSITE AND SOCIAL MEDIA CHANNELS: SIGN UP TO OUR MONTHLY NEWSLETTER HERE , AND FOLLOW US ONTWITTER @TREAT_NMD
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We're hosting our next Duchenne community webinar next Tuesday 21stApril at 12:30pm.
We want this webinar to be as useful as possible, so please submit any questions or requests for advice from the psychologists to support@duchenneuk.org by 10am tomorrow morning, Thursday 16th. Reply on Twitter 1250400390300008448Retweet
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TREAT-NMD FEATURED EVENTS POSTPONED – TREAT-NMD DMD EXPERT MASTERCLASS 2020 Please note this event has been postponed – further updates will be announced soon. We are pleased to announce that applications for the 2020 TREAT-NMD Expert Masterclass in Duchenne muscular dystrophy are now open. The event will take place in Sao Paulo, Brazil on 6th and 7th May 2020 and feature sessions on standards RARE DISEASE DAY 2020 Rare Disease Day 2020 is on Saturday 29th February! More information on how you can get involved is available on the dedicated website. 8TH DYSFERLIN CONFERENCE The Jain Foundation Dysferlin conference is the only meeting of its kind and focuses specifically on dysferlin and the related diseases caused by mutations in dysferlin. The Dysferlin conference includes presentations from leading international scientists on the most up to date information (mostly unpublished) regarding the state of dysferlin research, significant time for discussions andOPPORTUNITIES
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Do you want to receive the TREAT-NMD newsletter? * 6TH TREAT-NMD CONFERENCE 9-11TH DECEMBER 2019 We are delighted to announce that our 2019 conference was a complete sell out. It attracted over 300 delegates including academics, patients and carers, patient advocacy organisations, clinical specialists and industry. Please visit our dedicated Conference Website hereLINKS
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