News

Cell Therapy DT-DEC01 Showing Safety, Motor Gains in DMD Trial

DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle strength and motor function in the first three boys enrolled in a Phase 1 clinical trial. Based on these positive results of the therapy’s low dose, with follow-up ranging from one…

Vamorolone Filing for FDA Approval for DMD Expected by June

Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this year, with a filing expected to be completed by the end of June. The submission of the companies’ rolling new drug application (NDA) to the U.S. Food and Drug Administration…

Muscular Dystrophy Association Adds 13 New Care Centers in US

Thirteen new hospitals and health institutions have been added to the Muscular Dystrophy Association’s MDA Care Center Network, expanding the network’s reach across the United States. These additions, which comprise more than 150 care centers, include 10 new grant awards as well as three care affiliates that have been…

Electrical Stimulation That Mimics Lifting Weights May Benefit DMD

Electrical muscle stimulation that mimics resistance training exercises improves muscle structure, function, and survival rates in a Duchenne muscular dystrophy (DMD) zebrafish model, a new study reveals. “The longstanding consensus in the muscular dystrophy field is that minimizing resistance training preserves muscle strength and mass because it lowers the…

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…