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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…

The first patient has been dosed in a pilot study evaluating vamorolone as a potential treatment for Becker muscular dystrophy (BMD). The steroid medication has been under development by ReveraGen BioPharma as a possible therapy for other types of muscular dystrophy. Now, a Phase 2 trial…

Dyne Therapeutics’ FORCE platform delivered its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) directly to muscles in a mouse model of the disease, according to the results of a preclinical study. The potential DMD treatment improved dystrophin protein production and enhanced physical activity in treated mice. The…

Transferring a healthy version of the Large1 gene into adult mice with severe congenital muscular dystrophy restored the function of muscles involved in movement and breathing, a study found. The use of this gene therapy translated into an extended lifespan for the mice, which had congenital muscular dystrophy —…

The U.S. Food and Drug Administration (FDA) has given the go-ahead for the first-in-human dosing of Cure Rare Disease’s (CRD) investigational genome-editing therapeutic for Duchenne muscular dystrophy (DMD). Dosing of a single patient with the treatment, dubbed CRD-TMH-001, will happen soon at the University of Massachusetts Chan Medical…

Under an agreement with the Broad Institute, Sarepta Therapeutics has acquired an exclusive license to the gene therapy vector MyoAAV in potentially treating Duchenne muscular dystrophy (DMD) and several other neuromuscular and cardiac indications. MyoAAV is a new vector for delivering genetic therapies to cells, with evidence that…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant accelerated approval to the company’s experimental gene therapy SRP-9001 for people with Duchenne muscular dystrophy (DMD) who are able to walk. “We are delighted to confirm that based on the feedback we received…

Treatment with dystrophin expressing chimeric (DEC) cells was safe and led to long-term cellular and functional improvements in the heart, respiratory, and leg muscles of a mouse model of Duchenne muscular dystrophy, a study found. The findings support “future clinical application of DEC as a safer and potentially more…