In December 2020, CureDuchenne Ventures announced funding support for PepGen, Inc. to advance their Duchenne exon-skipping program to the clinic for trial. PepGen’s EDO cell-penetrating peptide platform is designed to improve the delivery of […]
Solid Biosciences issued a press release providing new, positive 2-year data from their IGNITE DMD clinical study in advance of the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. […]
**UPDATE** Duchenne gene therapy developers and scientific experts discuss AAV in DMD: Antitransgene SAEs. Please watch this webinar to learn more about recent concerns in gene therapy clinical trials. **ORIGINAL POST** […]
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 […]
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 […]
In this webinar, the NS Pharma team presents updates for the Duchenne community: Access programs for Viltepso, which is suitable for individuals with Duchenne who are amenable to exon 53 […]
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 […]
This presentation by Lauren Morgenroth, CGC and Ana Christensen, MPH at TRiNDS is part of the “A Holistic View of Clinical Trials” webinar and covers the following topics: the clinical […]
Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing […]
