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** […]
In 2015, CureDuchenne supplied early funding to Capricor Therapeutics in support of a novel, first-in-class therapy to improve cardiac function in people with Duchenne muscular dystrophy. Duchenne can cause debilitating […]
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 […]
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 […]
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 […]
CureDuchenne joins forces with NIH, FDA, and private organizations to increase effective gene therapies for rare diseases CureDuchenne is delighted to be a partner in the Accelerating Medicines Partnership Bespoke […]
