News

Santhera Pharmaceuticals reports that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for its drug Raxone (idebenone;  U.S. brand name, Catena) as a treatment for Duchenne muscular dystrophy (DMD) in patients with respiratory function decline and who are not under glucocorticoid therapy. The EMA validation confirms…

  Raul Carranza was born in Chula Vista 27 years ago. The recent graduate in political science from the University of California San Diego, and future law student,  is one of the university’s most heralded students. Diagnosed with muscular dystrophy (MD) at the age of two, and by his third birthday…

New research suggests that heart arrhythmia in myotonic dystrophy (DM) patients is caused by mutant RNA in the heart’s sodium channel. The study, which adds to the understanding of this symptom, was titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophy,” and published in …

CureDuchenne, a nonprofit group working to advance research and improve care for people with Duchenne muscular dystrophy (DMD), is partnering with Catabasis Pharmaceuticals to host a webinar discussing promising results from the first part of two-part MoveDMD clinical trial. The webinar is set for Wednesday, June 22, at 1 p.m. ET…

Northbrook, Illinois-based Marathon Pharmaceuticals reports it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the corticosteroid drug deflazacort as a treatment for Duchenne muscular dystrophy (DMD). There is currently no FDA-approved treatment or cure for DMD, the most common and severe form…

BioBridges, based in Wellesley Hills, Massachusetts, has announced its ongoing support for teen Emily Quitzau and her JettRide fundraiser in July. Emily Quitzau is a 14-year-old triplet with two brothers were both born with severe disabilities. Emily’s brother Calvin was diagnosed with Duchenne muscular dystrophy (DMD) when he was 5…

Catabasis Pharmaceuticals, in a joint initiative with Parent Project Muscular Dystrophy (PPMD), recently hosted the webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy.” The clinical-stage biopharma company, together with PPMD, a nonprofit organization founded in 1994 by parents of children with Duchenne and…

A new study from the University of Liverpool, has identified high amounts of a neutrophil-derived protein in the muscle of a Duchenne muscular dystrophy (DMD) mouse model. The finding shows that the protein hinders stem cells from repairing damaged muscle and it reveals a potential drug target for DMD therapies. The…

Recent research has revealed a new way of thinking about muscular dystrophy – and potential future drugs that could target laminin signaling to promote regeneration. Laminin, a protein present in the mesh-like structure surrounding cells called the extracellular matrix, determines if stem cells turn into new muscle, or fat cells. The study,…

Researchers at Johns Hopkins Medicine in Baltimore were able to generate muscle cells from skin cells of Duchenne muscular dystrophy (DMD) patients, forming human cell models of different DMD genetic mutations that will allow researchers to further understand the disease and test new therapies. The scientists were also able to correct…