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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 […]
CureDuchenne Commits $1M to Gennao Bio for Rare Muscle Disease Research
Investment to Expand First-in-Class, Nucleic Acid Therapeutics Towards Targeting Muscle Diseases Newport Beach, Calif. (December 22, 2021) – CureDuchenne Ventures, a subsidiary of CureDuchenne, a leading global nonprofit focused on […]
NS Pharma Pipeline Updates and Access to Viltepso
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 […]
New Developments in Mobile Arm Supports for Upper Extremity Weakness
Our presenters take us through the ins and out of mobile arm supports that are currently available, how they work, who they work for and other details. Many individuals living […]
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 […]
CureDuchenne Joins AMP BGTC
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 […]
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 […]