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Clinical Hold of DYNE-251
On January 14th 2022, the U.S. FDA placed a clinical hold on Dyne’s Investigational New Drug (IND) submission for the launch of their clinical trial of DYNE-251, which targets Duchenne […]
IGNITE DMD Phase I/II Clinical Trial Community Letter
Solid Biosciences provided an update on their programs this week. Notably, their microdystrophin Phase 1/2 trial continues to dose participants, and they are on track to report dystrophin levels and […]
Sarepta Announces Results From SRP-9001 Study
Sarepta today announced topline results from Part 2 of Study 102, an ongoing, randomized, double-blind, placebo-controlled clinical trial evaluating its investigational gene transfer therapy SRP-9001 in Duchenne patients. In […]
Early Phase Study of SRP-9001 in Boys with Duchenne
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy Sarepta is now expanding their trial of SRP-9001 (ENDEAVOR) to […]
A Message From Pfizer On Our DMD Clinical Program
We would like to share a recent change in our gene therapy clinical program for DMD. Three serious adverse events of muscle weakness, two of which involved myocarditis (inflammation of […]
Sarepta Therapeutics to Initiate Part B of MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Following Positive Interactions with FDA
– Company Anticipates Part B of MOMENTUM to Serve as Pivotal Study for SRP-5051 and to Seek Accelerated Approval if Successful – Ambulatory and Non-Ambulatory Patients Between the Ages of […]
CureDuchenne Ventures Early Funding Shows Promising Returns as FDA Fast Tracks Edgewise’s EDG-5506
CureDuchenne Ventures’ early funding of Edgewise in September of 2019 is returning more good news. In November of 2020, Edgewise began its Phase 1 clinical trial on EDG-5506, an experimental […]
Santhera and ReveraGen Announce Positive and Statistically Highly Significant Topline Results with Vamorolone in Pivotal VISION-DMD Study
June 1, 2021 VISION-DMD 24-week study readout: Update from Santhera Dear U.S. Duchenne community, We are happy to provide an update of the VISION-DMD Phase 2b study conducted by Santhera […]
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
Results from the first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus Treated patients achieved mean micro-dystrophin expression levels […]