Marisa Wexler, MS,  senior science writer—

Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a Wednesday webinar hosted by Parent Project Muscular Dystrophy (PPMD). PTC is the maker of Emflaza…

The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical care in muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders. The team consists of three physicians, all experts in these conditions and leading clinicians at an…

Because of the global COVID-19 pandemic, Italfarmaco has paused enrollment of boys with Duchenne muscular dystrophy (DMD) in a Phase 3 trial of its investigational treatment givinostat, the company said in a recent webinar hosted by Parent Project Muscular Dystrophy. Although treatment of current…

The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. The 30-minute session can be accessed on the MDA’s Facebook page, and starts at 6 p.m. EST. People with…

Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune system’s ability to work as it should, making it unlikely to raise a person’s COVID-19 risk, Catabasis Pharmaceuticals said. COVID-19 is believed to pose the greatest threat to older people and those…

Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as a treatment for facioscapulohumeral muscular dystrophy (FSHD). The data, “Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability and Target Engagement,” were presented by Michelle…

Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), both approved for the treatment of Duchenne muscular dystrophy (DMD), during the COVID-19 pandemic. Also, the company continues to work with regulatory authorities to guarantee the continuation…

Machine learning could be used to help diagnose specific forms of muscular dystrophy (MD) using magnetic resonance imaging (MRI) scans, a study suggests. The study, “Accuracy of a machine learning muscle MRI-based tool for the diagnosis of muscular dystrophies,” was published in the journal…

Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as a potential way to treat Duchenne muscular dystrophy (DMD). Capricor’s lead candidate for DMD is CAP-1002, which is composed of cardiosphere-derived cells (CDCs). These are cardiac progenitor cells, meaning they are able to…

A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential therapies and in establishing a biomarker. The observational study (NCT04202627) is now enrolling patients, ages 10 to 65, at…