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Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. An investigational therapy called AOC-1001 decreased levels of the toxic RNA molecule that causes…

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 (Â鶹Éç) 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 (Â鶹Éç). 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 (Â鶹Éç) has named Keisha Greaves, a fashion entrepreneur and neuromuscular disease advocate from Cambridge, Massachusetts, as its latest National Ambassador. “I have been involved with Â鶹Éç because muscular dystrophy is now a part of me,” Greaves said in an Â鶹Éç 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 (Â鶹Éç). Researchers said these results support Flavocoxid’s further investigation as a potential Â鶹Éç 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 (Â鶹Éç) 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 Â鶹Éç 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…