Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment SRP-5051 in people with Duchenne muscular dystrophy (DMD)Â who are amenable to exon 51 skipping. Results from part A of the…
Ray Jordan and Cyndi Segroves, a married couple with facioscapulohumeral muscular dystrophy (FSHD), haven’t let their disability get in the way of anything. They’ve traveled from their Arizona home to destinations around the world — from Hawaii and New…
The muscles of people with Duchenne muscular dystrophy (DMD) show profound impairment, with changes in fatty molecules and energy metabolism, as compared with those of their healthy peers, a small French study revealed. While previous studies have reported such metabolic changes in DMD patients, this was the first…
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. People with myotonic dystrophy type 2 (DM2) tend to be older at symptom onset than those with myotonic…
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. Women with limb girdle muscular dystrophy type R9 (LGMDR9) are not at a greater risk of pregnancy complications,…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
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. Once-weekly corticosteroid treatment was safe and well-tolerated in people with Becker muscular dystrophy (BMD) or limb-girdle muscular dystrophy…
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 (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…
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