Pfizer’s Letter to the Duchenne Community We are pleased to share the good news that several regulatory authorities have approved the re-start of our Phase 3 ambulatory trial (CIFFREO) for […]
We are pleased to share that Edgewise Therapeutics is sponsoring a natural history study in Becker muscular dystrophy, to better understand the course of disease and define endpoints to be […]
National Center of Neurology and Psychiatry and Nippon Shinyaku announce positive results for a Phase I/II study evaluating the efficacy and safety of their exon 44 skipping drug (NS-089/NCNP-02) Results […]
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 […]
