Young muscle stem cells in patients with Duchenne muscular dystrophy have shortened telomeres, causing these cells to be less able to build new muscle, according to new research. A full discussion of the results of this study, “Single Stem Cell Imaging and Analysis Reveals Telomere Length Differences in Diseased Human and Mouse…
A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital myotonic dystrophy develops over time. The three-year grant will allow Dr. Nicholas Johnson, an assistant professor of neurology, pediatrics, and pathology, to study what scientists call the natural history…
The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to the treatment before its regulatory approval. Raxone was included in EAMS after the Medicines and Healthcare Products Regulatory Agency (MHRA)…
A recent study has found that wearing hand braces while sleeping may help boys with Duchenne muscular dystrophy retain some of their muscle function. MORE: Watch what happens when children meet a man with muscular dystrophy The small clinical trial had eight boys with the condition who had impaired wrist extension…
Sarepta Therapeutics says its lead candidate therapy for exon 53 skipping, golodirsen, showed potential to treat Duchenne muscular dystrophy (DMD) in a first clinical trial of DMD patients. According to results of the Phase 1/2 clinical study, 4053-101 (NCT02310906), golodirsen significantly boosted dystrophin protein production in 25 boys with confirmed deletions…
PTC Therapeutics recently announced the recipients of the 2017 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) Awards program, recognizing excellence and innovation in the Duchenne muscular dystrophy (DMD) community. The 2017 awards were on World Duchenne Awareness Day, on Sept. 7. The STRIVE program was launched…
Parent Project Muscular Dystrophy is holding an awareness, fund-raising and educational campaign throughout September to fight Duchenne muscular dystrophy (DMD). It has scheduled events every day of Duchenne Action Month. You’ll find a calendar of activities here. September is traditionally a busy month for the worldwide DMD community.
In this video from Gawker, Rich Juzwiak talks to comedian Steve Way to find out how having muscular dystrophy affects his life and how it impacts his comedy. MORE:Â Cyclists ride from Seattle to New York for facioscapulohumeral muscular dystrophy Way talks about the misconceptions…
These videos show how people living with muscular dystrophy (MD) can smash the stereotypes associated with having a disability, and lead rich and full lives. First up is 35-year-old Michael Klinkhamer, who has Becker muscular dystrophy. In this video shared by Story Hive, Michael explains that he spends all of…
In this super cute video from WatchCut Video, children of various ages meet a man living with muscular dystrophy and ask some candid questions about his life with the condition. MORE: Talking to family members about your child’s neuromuscular disorder Amine talks about how having…
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