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 is happy to share the news that REGENXBIO will be launching a novel gene therapy trial in Duchenne during the first half of 2022. RGX-202 aims to deliver a […]
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 […]
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 […]
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 […]
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 […]
– 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 […]
In WMS 2021’s final day and “late-breaking news” session, Capricor Therapeutics announced positive final data from its Phase 2 HOPE-2 clinical trial in Duchenne patients treated with CAP-1002. HOPE-2 was […]
