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 […]
The 2021 National Organization for Rare Disorders (NORD) Breakthrough Summit saw a number of innovations unveiled for the rare disease community. RareDisease Advisor was there to cover the highlights of […]
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 […]
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 […]
