The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
The DuchenneXchange online social platform has launched a mobile and desktop app for the Duchenne muscular dystrophy (DMD) community, to encourage discussion and to share news, diagnosis and treatment resources, and clinical trial information. The goal for the new app is to make the…
The U.S. Food and Drug Administration has granted fast track designation to pamrevlumab, an investigational treatment for Duchenne muscular dystrophy (DMD). The designation is given to medications that have the potential to treat serious conditions and to fill an unmet medical need. The designation allows the therapy’s developer, FibroGen,…
The Muscular Dystrophy Association (MDA) has named Keisha Greaves, a fashion entrepreneur and neuromuscular disease advocate from Cambridge, Massachusetts, as its latest National Ambassador. “I have been involved with MDA because muscular dystrophy is now a part of me,” Greaves said in an MDA press release. “I…
Following its acquisition last year by Astellas Pharma, Audentes Therapeutics now has become Astellas Gene Therapies. Named a Gene Therapy Center of Excellence, it will work to advance the development of potentially life-changing gene therapies for several conditions, including muscular dystrophy (MD). “The field of gene therapy has…
Flavocoxid was well-tolerated and lessened inflammation in a small clinical trial involving boys with Duchenne muscular dystrophy (DMD). Researchers said these results support Flavocoxid’s further investigation as a potential DMD therapy. The findings were published in the journal Brain Sciences, in “A Phase 1/2…
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
FibroGen has begun a second Phase 3 clinical trial of pamrevlumab, given with corticosteroids, among boys with Duchenne muscular dystrophy (DMD) who are able to walk. The trial, LELANTOS-2 (NCT04632940), is enrolling approximately 70 participants ages 6 to 12 at three U.S. sites — Arkansas…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Long-term treatment with the experimental steroid therapy vamorolone safely and effectively delays motor function decline in boys with…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy PF-06939926 continues to demonstrate an acceptable safety profile and the potential for…
Get regular updates to your inbox.