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A first patient has been dosed in Denmark as part of a Phase 1/2 trial of ATA-100, Atamyo Therapeutics’ one-time gene therapy for a specific form of limb-girdle muscular dystrophy called LGMD-2I or LGMD-R9. “This is an exciting milestone for our company but most importantly, if this clinical trial is…

The beginning of a new college football season also means it’s time for Coach to Cure MD (CTCMD) — a campaign aimed at calling attention to Duchenne muscular dystrophy (DMD) and raising research funds for a cure. Slated for Sept. 24, the 15th annual event is a…

The Muscular Dystrophy Association (MDA) has awarded more than $17 million to projects aimed at advancing research and developing new treatments in muscular dystrophy (MD) and other neuromuscular diseases. The funding adds to the more than $1 billion the MDA has already invested in research. “The Muscular Dystrophy…

Four months of treatment with the experimental oral therapy EDG-5506 reduced markers of muscle damage and improved measures of physical function among men with Becker muscular dystrophy (BMD) in the Phase 1 ARCH clinical trial, new data show. Edgewise Therapeutics, the company developing EDG-5506, recently launched a Phase…

The first patient has received Dyne Therapeutics‘ investigational therapy DYNE-251 for treating Duchenne muscular dystrophy (DMD), the company has announced. The therapy is being tested in the DELIVER Phase 1/2 clinical trial and is indicated for patients with mutations amenable to exon 51 skipping. “We are excited to…

Vamorolone, an investigational therapy for Duchenne muscular dystrophy (DMD), maintained biomarkers of bone formation and turnover compared to prednisone, according to the now published details of the VISION-DMD study. Standard anti-inflammatory corticosteroids, such as prednisone, have been shown to stunt growth with long-term use. Vamorolone is a…

Treatment with Exondys 51 (eteplirsen) outperformed standard of care therapy at delaying respiratory decline in boys with Duchenne muscular dystrophy (DMD), an analysis of data from several clinical trials found. Compared with boys on other therapies, those treated with Exondys 51 in trials had “a delay of…

For the fourth year, a family in Montana is hosting “Calves to Cure,” a cattle auction that aims to raise funds to support developing new treatments, or even a cure, for Duchenne muscular dystrophy. The event will take place Sept. 15 in Billings, Montana. All proceeds from the…

Note: This story was updated Aug. 31, 2022, to clarify that MDA Advocate Madison Lawson will throw out the honorary pitch at the Cardinals game alongside Donald S. Wood. Supporters across the country are poised to mark National Muscular Dystrophy Awareness Month, observed each September in the U.S. The…

Children with myotonic dystrophy type 1 (DM1) showed a decline in cognitive function over two years, with frequent impairment in visuospatial skills and attention, a small study suggested. The data indicate cognitive, neuropsychological, emotional, and behavioral assessments should be administered to children with DM1 periodically during development, the researchers…