Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. “We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for ... May 12, 2023 · SRP-9001, otherwise known as delandistrogene moxeparvovec, is designed to deliver a copy of a gene encoding microdystrophin to muscle cells using a lab-engineered, harmless virus as a vector. It is prepared as a suspension of concentration of 1.33 x 1013 vg/mL, and is supplied in a single-use, 10 mL vial for a single-dose, intravenous infusion. Nov 29, 2022 · SRP-9001 is designed to deliver a copy of a gene encoding micro-dystrophin — a shortened but functional version of the dystrophin protein, whose lack causes DMD — to muscle cells using a lab-engineered, harmless virus as a vector. Jul 6, 2022 · SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche. Data are being presented this week at the 17th International Congress on Neuromuscular Diseases ... Oct 5, 2021 · SRP-9001 is one of the leading investigational gene therapies that is being developed for treating DMD and the EMBARK study is the first pivotal study to evaluate any gene therapy for the indication. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. Nov 28, 2022 · The BLA is supported by efficacy and safety data from the SRP-9001-101, SRP-9001-102, and SRP-9001-103 studies (ClinicalTrials.gov Identifier: NCT03375164, NCT03769116, NCT04626674, respectively ... A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD) The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. Aug 10, 2022 · BLA filing of SRP-9001 for the treatment of patients with DMD is expected in fall of 2022; If filing and review go well then FDA Accelerated approval possible mid-2023. Mar 23, 2023 · A set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the investigational gene therapy delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) in the treatment of Duchenne muscular dystrophy (DMD) has been established by the Delphi panel, which reviewed data from 3 clinical trials of the therapy. 1 Nov 28, 2022 · SRP-9001 has demonstrated safety and efficacy in over 80 treated patients so far, with follow-up of up to 4 years post-treatment. The most recent data were presented from the ENDEAVOR study (Study SRP-9001-103; NCT04626674) in July 2022 and demonstrated an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines in 20 participants at 52 weeks, as well ... Aug 23, 2023 · ³ Other LGMD targets in development: SRP-9005 (LGMD2C/R5 γ-sarcoglycan), SRP-9006 (LGMD2L/R12 Anoctamin 5), and SRP-9010 (LGMD2A/R1) Download PDF of Our Pipeline For more information about our pipeline, please contact Sarepta's Patient Affairs team at [email protected] . Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the ... Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... Mar 23, 2023 · A set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the investigational gene therapy delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) in the treatment of Duchenne muscular dystrophy (DMD) has been established by the Delphi panel, which reviewed data from 3 clinical trials of the therapy. 1 May 11, 2023 · SRP-9001 is designed to deliver to muscle cells a gene encoding micro-dystrophin, a shortened version of the dystrophin protein, which is a protein that is lacking in DMD patients. Sarepta’s application is specifically based on data suggesting the therapy can increase micro-dystrophin levels as designed. Mar 23, 2023 · A set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the investigational gene therapy delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) in the treatment of Duchenne muscular dystrophy (DMD) has been established by the Delphi panel, which reviewed data from 3 clinical trials of the therapy. 1 Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... Apr 10, 2023 · SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of functional components of dystrophin. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving ... Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. Jun 22, 2023 · The FDA has approved Sarepta Therapeutics’ investigational gene therapy SRP-9001, otherwise known as delandistrogene moxeparvovec, for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Jul 6, 2022 · In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=<0.0001) Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. “We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for ... Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the ... Nov 28, 2022 · SRP-9001 has demonstrated safety and efficacy in over 80 treated patients so far, with follow-up of up to 4 years post-treatment. The most recent data were presented from the ENDEAVOR study (Study SRP-9001-103; NCT04626674) in July 2022 and demonstrated an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines in 20 participants at 52 weeks, as well ... Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... Nov 28, 2022 · The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. Extensive pre-clinical evidence also formed part of the BLA. Apr 22, 2021 · SRP-9001 is an adeno-associated virus (AAV) mediated gene therapy that delivers a micro-dystrophin-encoding gene to the muscles and is intended as a curative treatment. The specific vector deployed in the gene transfer, AAVrh74, has been shown to achieve the efficient delivery of micro-dystrophin to skeletal muscle with tolerable immunogenicity ... Jan 4, 2021 · A natural history comparison cohort was drawn retrospectively from the ImagingDMD study. We identified 54 participants who had a total of 110 study visits (between 2011 and 2018) aged between 4.9 and 7.9 years, matching the age of the participants who received SRP-9001. All participants in the natural history cohort were treated with ... We would like to show you a description here but the site won’t allow us. Jan 11, 2022 · Participants in the trial will be given a single infusion of the gene therapy or placebo and be monitored for 52 weeks (about a year). Then, participants originally given SRP-9001 will get an infusion of placebo, and vice versa, followed by another year of monitoring. The study’s main goal is to measure changes in NSAA scores. Nov 30, 2022 · According to a recent announcement, the FDA accepted and granted priority review to Sarepta Therapeutics ’ biologics license application (BLA) for SRP-9001, an investigative gene therapy for treating Duchenne muscular dystrophy (DMD), with a PDUFA date set for May 29, 2023. 1. "We are delighted to announce that the FDA has accepted Sarepta ... Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. May 3, 2022 · Patients treated with SRP-9001 generally maintained muscle strength (Time to Rise and 4-Stair Climb) and showed improvement in ambulation ability (100m) from baseline to Year 3. Conclusions: The observed safety profile and the enduring response following gene transfer provides proof-of-concept for the continuation of clinical trials assessing ... Mar 18, 2023 · In advance of the upcoming regulatory action deadline for Sarepta Therapuetics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (also known as SRP-9001), the FDA has determined it will hold an advisory committee (AdComm) meeting. 1 The current Prescription Drug User Fee Act action date for the treatment’s biologics license ... Jan 5, 2023 · Ms. Borland discusses SRP-9001’s mechanism of action in terms of its 3 essential components, its vector, promoter, and transgene. She begins by explaining that SRP-9001 uses AAVrh74 as a vector, which was selected for its tissue selectivity for skeletal and cardiac muscle, as well as its low potential for pre-existing antibodies. Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. The company noted that SRP-9001 would be the first gene therapy for Duchenne. Sarepta is ... Jul 7, 2022 · Twenty participants in SRP-9001-103 (ENDEAVOR, NCT04626674) treated with SRP-9001 showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control ... Jun 22, 2023 · For SRP-9001, the surrogate endpoint is a gene called micro-dystrophin, which codes for a shortened form of the dystrophin protein, measured 12 weeks after treatment with SRP-9001. In the US, there are four drugs which already have accelerated access for DMD, three of which are Sarepta’s, and all of which are exon skipping treatments. Aug 2, 2022 · Equally impressive SRP-9001 treated patients improved 3.8 points on unadjusted means and 3.2 requiring of 52 weeks on NSAA compared to the propensity Master external control group, with a p-value ... May 12, 2023 · SRP-9001 dystrophin protein expression is reasonably likely to predict clinical benefit (product design, biological, and empirical data showing direct change in underlying pathophysiology of... Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... Sep 29, 2022 · SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of functional components of dystrophin. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving ... Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... We would like to show you a description here but the site won’t allow us. Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. Mar 18, 2023 · An additional integrated analysis of 52 patients across studies SRP-9001-101, SRP-9001-102, and ENDEAVOR also showed that at 1 year, patients treated with SRP-9001 at the target dose improved 3.1 points (unadjusted means) and 2.4 points (least squared means) on NSAA versus propensity-weighted external control (P <.0001). 2. REFERENCES 1 ... A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD) The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. May 3, 2022 · Patients treated with SRP-9001 generally maintained muscle strength (Time to Rise and 4-Stair Climb) and showed improvement in ambulation ability (100m) from baseline to Year 3. Conclusions: The observed safety profile and the enduring response following gene transfer provides proof-of-concept for the continuation of clinical trials assessing ... Nov 28, 2022 · SRP-9001 has demonstrated safety and efficacy in over 80 treated patients so far, with follow-up of up to 4 years post-treatment. The most recent data were presented from the ENDEAVOR study (Study SRP-9001-103; NCT04626674) in July 2022 and demonstrated an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines in 20 participants at 52 weeks, as well ... May 18, 2021 · About SRP-9001-103 (ENDEAVOR) Study SRP-9001-103 (Study 103) is an open-label clinical trial of SRP-9001 that has enrolled 20 participants with Duchenne muscular dystrophy between the ages of 4-7. May 12, 2023 · SRP-9001 dystrophin protein expression is reasonably likely to predict clinical benefit (product design, biological, and empirical data showing direct change in underlying pathophysiology of... Sarepta’s gene therapy engine is tailored to specific disease states and aims to select individual components that target specific tissues and cells with the goal of optimizing expression of the select protein in those tissues. Together, these three components form what is called a construct . Nov 28, 2022 · SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The SRP-9001 development program is sponsored by Sarepta Therapeutics, in partnership with Roche, and has been partially funded by PPMD. Jan 4, 2021 · A natural history comparison cohort was drawn retrospectively from the ImagingDMD study. We identified 54 participants who had a total of 110 study visits (between 2011 and 2018) aged between 4.9 and 7.9 years, matching the age of the participants who received SRP-9001. All participants in the natural history cohort were treated with ... May 11, 2023 · SRP-9001 is designed to deliver to muscle cells a gene encoding micro-dystrophin, a shortened version of the dystrophin protein, which is a protein that is lacking in DMD patients. Sarepta’s application is specifically based on data suggesting the therapy can increase micro-dystrophin levels as designed. Mar 18, 2023 · In advance of the upcoming regulatory action deadline for Sarepta Therapuetics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (also known as SRP-9001), the FDA has determined it will hold an advisory committee (AdComm) meeting. 1 The current Prescription Drug User Fee Act action date for the treatment’s biologics license ... Oct 11, 2021 · In new analyses presented at “SRP-9001 Micro-dystrophin Day,” results from participants treated with SRP-9001 in Study SRP-9001-101 (n=4, ages 4 to 7) found that participants in Study 101 improved 8.6 points on the North Star Ambulatory Assessment (NSAA)* compared to a matched natural history cohort three years following a single ... Jan 7, 2021 · Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 102 uses clinical process SRP-9001 and has two primary endpoints: micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks ... May 11, 2023 · SRP-9001 is designed to deliver to muscle cells a gene encoding micro-dystrophin, a shortened version of the dystrophin protein, which is a protein that is lacking in DMD patients. Sarepta’s application is specifically based on data suggesting the therapy can increase micro-dystrophin levels as designed. Mar 23, 2023 · A set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the investigational gene therapy delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) in the treatment of Duchenne muscular dystrophy (DMD) has been established by the Delphi panel, which reviewed data from 3 clinical trials of the therapy. 1 Oct 4, 2022 · SRP-9001 is designed to deliver a gene encoding micro-dystrophin (a shortened but functional version of the dystrophin protein) to the body’s muscle cells using a viral vector. The therapy is being co-developed by Sarepta and Roche. “If approved, SRP-9001 will be the first gene therapy available for Duchenne patients,” Ingram said. May 12, 2023 · In documents released Wednesday, FDA reviewers concluded Sarepta’s clinical trials “do not provide unambiguous evidence” that the therapy, known as SRP-9001, is “likely beneficial for ... Nov 28, 2022 · The BLA is supported by efficacy and safety data from the SRP-9001-101, SRP-9001-102, and SRP-9001-103 studies (ClinicalTrials.gov Identifier: NCT03375164, NCT03769116, NCT04626674, respectively ... Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. The company noted that SRP-9001 would be the first gene therapy for Duchenne. Sarepta is ... Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. “We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for ... Jun 22, 2023 · SRP-9001 is a novel DMD gene-replacement therapy co-invented by Dr. Mendell and Louise Rodino-Klapac, PhD, formerly at Nationwide Children’s and currently the executive vice president, head of Research and Development, and chief scientific officer at Sarepta Therapeutics. Jan 5, 2023 · Ms. Borland discusses SRP-9001’s mechanism of action in terms of its 3 essential components, its vector, promoter, and transgene. She begins by explaining that SRP-9001 uses AAVrh74 as a vector, which was selected for its tissue selectivity for skeletal and cardiac muscle, as well as its low potential for pre-existing antibodies. Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. “We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for ... May 12, 2023 · In documents released Wednesday, FDA reviewers concluded Sarepta’s clinical trials “do not provide unambiguous evidence” that the therapy, known as SRP-9001, is “likely beneficial for ... Dec 23, 2019 · SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. “As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... Mar 18, 2023 · An additional integrated analysis of 52 patients across studies SRP-9001-101, SRP-9001-102, and ENDEAVOR also showed that at 1 year, patients treated with SRP-9001 at the target dose improved 3.1 points (unadjusted means) and 2.4 points (least squared means) on NSAA versus propensity-weighted external control (P <.0001). 2. REFERENCES 1 ... Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... Nov 28, 2022 · SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. “We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for ... Aug 2, 2022 · Equally impressive SRP-9001 treated patients improved 3.8 points on unadjusted means and 3.2 requiring of 52 weeks on NSAA compared to the propensity Master external control group, with a p-value ... Jan 7, 2021 · About SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ... We would like to show you a description here but the site won’t allow us.
May 12, 2023 · SRP-9001 uses viruses to ferry a copy of a gene to muscles to help make up for one that’s causing the disease. Hennick and many other parents like her advocated for the treatment’s accelerated... . Faith build demon
May 12, 2023 · The vote, 8 to 6, came after a day of testimony from speakers for Sarepta Therapeutics, the maker of the gene therapy called SRP-9001, FDA scientists and families whose children have Duchenne ... Jun 27, 2023 · Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy Nov 28, 2022 Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta’s Gene Therapy for the ... Jan 4, 2021 · A natural history comparison cohort was drawn retrospectively from the ImagingDMD study. We identified 54 participants who had a total of 110 study visits (between 2011 and 2018) aged between 4.9 and 7.9 years, matching the age of the participants who received SRP-9001. All participants in the natural history cohort were treated with ... May 3, 2022 · Patients treated with SRP-9001 generally maintained muscle strength (Time to Rise and 4-Stair Climb) and showed improvement in ambulation ability (100m) from baseline to Year 3. Conclusions: The observed safety profile and the enduring response following gene transfer provides proof-of-concept for the continuation of clinical trials assessing ... Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... Dec 23, 2019 · SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. “As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. Mar 18, 2023 · In advance of the upcoming regulatory action deadline for Sarepta Therapuetics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (also known as SRP-9001), the FDA has determined it will hold an advisory committee (AdComm) meeting. 1 The current Prescription Drug User Fee Act action date for the treatment’s biologics license ... Mar 23, 2023 · A set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the investigational gene therapy delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) in the treatment of Duchenne muscular dystrophy (DMD) has been established by the Delphi panel, which reviewed data from 3 clinical trials of the therapy. 1 May 3, 2022 · Patients treated with SRP-9001 generally maintained muscle strength (Time to Rise and 4-Stair Climb) and showed improvement in ambulation ability (100m) from baseline to Year 3. Conclusions: The observed safety profile and the enduring response following gene transfer provides proof-of-concept for the continuation of clinical trials assessing ... Jan 7, 2021 · Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 102 uses clinical process SRP-9001 and has two primary endpoints: micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks ... Apr 19, 2023 · Study SRP-9001-103 showed that SRP-9001-treated patients (n=20, ages 4 to 7) had significant improvements in functional motor abilities one year after treatment compared to an external control ... May 12, 2023 · SRP-9001 uses viruses to ferry a copy of a gene to muscles to help make up for one that’s causing the disease. Hennick and many other parents like her advocated for the treatment’s accelerated... Nov 28, 2022 · SRP-9001 has demonstrated safety and efficacy in over 80 treated patients so far, with follow-up of up to 4 years post-treatment. The most recent data were presented from the ENDEAVOR study (Study SRP-9001-103; NCT04626674) in July 2022 and demonstrated an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines in 20 participants at 52 weeks, as well ... Jan 7, 2021 · SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene, to muscle tissue, needed for muscle membrane stability. Jan 7, 2021 · Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 102 uses clinical process SRP-9001 and has two primary endpoints: micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks ... We would like to show you a description here but the site won’t allow us. .