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ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1INVESTOR RELATIONS
The Investor Relations website contains information about Sarepta Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.PRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
EXON 44 | SAREPTA CORPORATE REBUILD 2021 Sarepta Therapeutics All rights reserved. Footer Utility Nav. Privacy Policy; Terms of Use; EU-U.S. and Swiss-U.S. Privacy ShieldPolicy
LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sarepta’s senior vice president, gene therapy. She has led the design of most of Sarepta’s late-stage gene therapy candidates, has built and led Sarepta’s Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS This section of our website may contain dated or archived information which should not be considered current and may no longer be accurate. For current information, you are encouraged to review our most recent official corporate documents on file with the U.S. Securities and Exchange Commission. SAREPTA THERAPEUTICS CELEBRATING OUR PRIDE. At Sarepta, we strive to promote diversity, inclusion, equal opportunity and personal development across the organization. Our Employee Resource Group Pride created by employees who identify as LGBTQIA+ along with their allies provides members with a forum to promote a dynamic, welcoming, and friendly atmosphere while strengthening diversity and inclusion at Sarepta. SRP-9005 (LGMD2C Γ-SARCOGLYCAN) 2021 Sarepta Therapeutics All rights reserved. Footer Utility Nav. Privacy Policy; Terms of Use; EU-U.S. and Swiss-U.S. Privacy ShieldPolicy
ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1INVESTOR RELATIONS
The Investor Relations website contains information about Sarepta Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.PRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
EXON 44 | SAREPTA CORPORATE REBUILD 2021 Sarepta Therapeutics All rights reserved. Footer Utility Nav. Privacy Policy; Terms of Use; EU-U.S. and Swiss-U.S. Privacy ShieldPolicy
LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sarepta’s senior vice president, gene therapy. She has led the design of most of Sarepta’s late-stage gene therapy candidates, has built and led Sarepta’s Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS This section of our website may contain dated or archived information which should not be considered current and may no longer be accurate. For current information, you are encouraged to review our most recent official corporate documents on file with the U.S. Securities and Exchange Commission.PIPELINE & PRODUCTS
At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of development—many already in late-stage clinical trials. In many cases, development is being accelerated by our gene therapy engine, which potentially provides a more efficient model for drug design. DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females.CLINICAL TRIALS
Brief Summary: This study will be comprised of 2 parts: Part 1 will be conducted to evaluate the safety and tolerability of two doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 8 patients; Part 2 will be conducted for the selection of a high dose (high dose level 1 vs high dose level 2) (dose finding phase), and its comparison with the 30 mg/kg dose of eteplirsenINVESTOR RELATIONS
“Safe Harbor” Statement under the Private Securities Litigation Reform Act of 1995: The statements that are not historical facts contained in releases or in our posted events and presentations are forward-looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testingDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sarepta’s mission: “We know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. We’re in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.” GRANT AND SPONSORSHIP APPLICATION PORTAL Instructions for Sarepta’s Grant & Sponsorship Application Portal 5 of 32 8. After selecting an application type, you will be navigated to an Instructional Page, please read SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051 CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the 4045-301 CLINICAL TRIAL 4045-301 CLINICAL TRIAL For Duchenne muscular dystrophy (DMD) patients with deletion mu tations amenable to exon 45 or exon 53 skipping Information on site locations and MOMENTUM | CLINICAL TRIALS Multiple means that all participants will receive doses of the study drug once every 4 weeks throughout the study.Ascending dose means that there are different study drug dose levels in Part A that increase from one dose group to the next.All participants will eventually move to the same dose level, the maximum tolerated dose, in Part B of thestudy.
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine. DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SRP-9005 (LGMD2C Γ-SARCOGLYCAN) SRP-9005 (LGMD2C γ-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugPRESS RELEASES
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635 (c) (4) 5/18/21. Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta’s Commercial Process Material. 5/17/21. A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine. DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SRP-9005 (LGMD2C Γ-SARCOGLYCAN) SRP-9005 (LGMD2C γ-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugPRESS RELEASES
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635 (c) (4) 5/18/21. Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta’s Commercial Process Material. 5/17/21. A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
PIPELINE & PRODUCTS
Pipeline & Products | Sarepta Therapeutics. At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of development—many already in late-stage clinical trials. In many cases, development is being accelerated by our gene NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugCLINICAL TRIALS
Brief Summary: This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy GENE EDITING RESEARCH At Sarepta, we are committed to exploring every modality of precision genetic medicine that may help advance our mission to rescue lives otherwise impacted or stolen by rare disease. We believe gene editing, although early, has the potential to revolutionize the treatment of ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
SAREPTA THERAPEUTICS’ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
LIMB GIRDLE MUSCULAR DYSTROPHY (LGMD) Limb-girdle muscular dystrophy (LGMD) is a group of distinct diseases that cause weakness and wasting of the muscles, generally starting with the muscles around the hips and shoulders and eventually progressing to the arms and legs. However, some subtypes start distally at the leg or arm muscles and then progress to the hip andshoulder muscles.
MOMENTUM | CLINICAL TRIALS Participant Eligibility Criteria. Your child may be eligible to participate in Momentum trial if he meets the following eligibility criteria: Ambulatory and non-ambulatory boys with DMD who are 7-21 years old (patients who enroll in Part A) or 4-21 years old (patients who enroll in Part B) Has a genetic diagnosis of DMD and anout-of-frame
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy SRP-9005 (LGMD2C Γ-SARCOGLYCAN) SRP-9005 (LGMD2C γ-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sarepta’s mission: “We know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. We’re in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.” LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sarepta’s senior vice president, gene therapy. She has led the design of most of Sarepta’s late-stage gene therapy candidates, has built and led Sarepta’s Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and hasPRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy SRP-9005 (LGMD2C Γ-SARCOGLYCAN) SRP-9005 (LGMD2C γ-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment •The NSAA is only given to patients who are ambulant, or able to walk on their own.2 • The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a person’s functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sarepta’s mission: “We know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. We’re in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.” LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sarepta’s senior vice president, gene therapy. She has led the design of most of Sarepta’s late-stage gene therapy candidates, has built and led Sarepta’s Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and hasPRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C ‘written response only’ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugCLINICAL TRIALS
Brief Summary: This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophyINVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), CharcotDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sarepta’s mission: “We know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. We’re in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.” DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICS’ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
GRANT AND SPONSORSHIP APPLICATION PORTAL Instructions for Sarepta’s Grant & Sponsorship Application Portal 5 of 32 8. After selecting an application type, you will be navigated to an Instructional Page, please read SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051 CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the SRP-9004 (LGMD2D Α-SARCOGLYCAN) SRP-9004 (LGMD2D α-sarcoglycan) | Sarepta Corporate Rebuild. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. 4045-301 CLINICAL TRIAL 4045-301 CLINICAL TRIAL For Duchenne muscular dystrophy (DMD) patients with deletion mu tations amenable to exon 45 or exon 53 skipping Information on site locations and Skip to main contentPRESS RELEASES
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05/06/2020 SAREPTA THERAPEUTICS ANNOUNCES FIRST QUARTER 2020 FINANCIAL RESULTS AND RECENT CORPORATE DEVELOPMENTS*
04/30/2020 SAREPTA THERAPEUTICS ANNOUNCES INDUCEMENT GRANTS UNDER NASDAQ LISTING RULE 5635(C)(4)*
04/29/2020 SAREPTA THERAPEUTICS TO ANNOUNCE FIRST QUARTER 2020 FINANCIAL RESULTS AND RECENT CORPORATE DEVELOPMENTS ON MAY 6, 2020UTILITY MENU
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SOME SEE SLOW AND STEADY SCIENTIFIC PROGRESS. WE SEE A REVOLUTION. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy manufacturing capacity, and rethinking access and reimbursement models for revolutionary new treatments. We are in a daily race to transform genetic understanding into genetic medicine. Because every day is an opportunity to change the lives of people living with rare disease.Learn more
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SOME THINK A CURE FOR A RARE DISEASE IS A MIRACLE. OUR SCIENTISTS WOULD DISAGREE. Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA technologies, and gene editing. We are in a daily race to save lives stolen or impacted by rare disease.Learn more here
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PATIENTS CAN’T WAIT FOR THE NEXT BREAKTHROUGH IN MEDICAL RESEARCH.SO NEITHER WILL WE.
While there may be as many as 7,000 rare diseases, only a small percentage have treatments. That is why we are relentless in our dedication: Leverage the best science to help as many patients as possible. Today, we are doing just that in Duchenne muscular dystrophy, in six forms of limb-girdle muscular dystrophy, in Charcot-Marie-Tooth disease, and in Mucopolysaccharidosis type IIIA (MPS IIIA), among others. Sarepta will always follow the science and continuously evaluate other diseases and modalities to pursue.Learn more
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THE OPPORTUNITY TO TRANSFORM LIVES IS BREATHTAKING.JOIN US.
We see endless possibilities for changing the future of medicine. We’re looking for people who see unlimited potential in themselves, opportunity where others see roadblocks, and solutions limited only by the power of imagination and the drive to do things differently. Learn more about working at Sarepta* 1
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SAREPTA’S RESPONSE TO COVID-19 Read our Company Statement (Mar. 19,2020)
FOR PATIENTS IN THE U.S. TAKING AN APPROVED SAREPTA THERAPY, please contact SareptAssist at +1-888-727-3782 with anyquestions.
FOR PARTICIPANTS IN SAREPTA-SPONSORED CLINICAL TRIALS, Sarepta is working closely with your site teams to mitigate disruptions caused by COVID-19 and we encourage you to contact your site team to understand the status of your upcoming visits. The Sarepta team is committed to helping you navigate disruptions in your trial participation and can be reached at +1-800-690-2003 or via email clinicaltrials@sarepta.com to answer any questions or concerns during this uncertain time. RESOURCES FOR PATIENTS AND CAREGIVERSLearn More
AN OPPORTUNITY TO CHANGE THE FACE OF HEALTHCARE Sarepta President and CEO, Doug Ingram, addresses the audience of more than 650 healthcare partners gathered at the 2019 Express Scripts Outcomes Conference. The Annual Outcomes Conference brings the perspectives of innovators and payers together to catalyze the realization of healthcare’s full potential.PRESS RELEASES
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05/06/2020 SAREPTA THERAPEUTICS ANNOUNCES FIRST QUARTER 2020 FINANCIAL RESULTS AND RECENT CORPORATE DEVELOPMENTS*
04/30/2020 SAREPTA THERAPEUTICS ANNOUNCES INDUCEMENT GRANTS UNDER NASDAQ LISTING RULE 5635(C)(4)*
04/29/2020 SAREPTA THERAPEUTICS TO ANNOUNCE FIRST QUARTER 2020 FINANCIAL RESULTS AND RECENT CORPORATE DEVELOPMENTS ON MAY 6, 2020 SPOTLIGHT ON: GENE THERAPIES IN DEVELOPMENT GENE THERAPY MANUFACTURING Dr. Reed Clark, a pioneering expert on gene therapy process development and AAV biology, discusses the complexities and challenges of gene therapy manufacturing and Sarepta's manufacturing approach. EXPLORING THE POTENTIAL OF GENE THERAPIES FOR LIMB-GIRDLE MUSCULARDYSTROPHIES
Dr. Louise Rodino-Klapac, discusses the science of limb-girdle muscular dystrophies (LGMDs) and how our gene therapy engine will help engineer potential therapies for this group of inherited neuromuscular diseases.CORPORATE UPDATES
A NEW FDA-APPROVED TREATMENT View our approved productsRead press release
SAREPTA THERAPEUTICS' PRESENTATION FROM THE 38TH ANNUAL J.P. MORGAN HEALTHCARE CONFERENCE (2020) View presentation (PDF)*
Sarepta Therapeutics @SareptaTwitter Logo On today’s #Q12020Resultscall, CEO, Doug
Ingram, expressed pride and appreciation for SRPT team members, including our facility-dependent colleagues, who continue to advance our mission for those living with #rarediseaseduring the #COVID19
pandemic. bit.ly/3baeaHl* Reply
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6 May 2020
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Sarepta Therapeutics @SareptaTwitter Logo On our #Q12020Results call happening now, SRPT CEO, Doug Ingram, emphasizes the mission to transform lives that drives us – in the midst of the #COVID19 crisis – and every day. Access the live webcast: bit.ly/2zj7cmo* Reply
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6 May 2020
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Sarepta Therapeutics @SareptaTwitter Logo Reminder: Forward-looking statements are subject to risks, including those described in SRPT's #Q12020Results and corporate update #PressRelease Tune in here: bit.ly/2zj7cmo* Reply
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