News

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…

Researchers at the University of Liverpool in England reported that people with muscular dystrophy (MD) may have an enzyme in their muscle cells that stops the ability of these cells to repair themselves. The report, “Elastase levels and activity are increased in dystrophic muscle and…

BioMarin Pharmaceutical Inc. has announced the withdrawal of its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The decision follows deliberations at the May 2016 Committee for Medicinal Products for…

Ryan Benton, the first patient to be approved in the United States for allogenic adult stem cell transplantation as a Duchenne muscular dystrophy (DMD) treatment, turned 30 this year — largely exceeding his original life expectancy. DMD is a genetic disease that affects 1 out of every 3,600 male infants, leading to…

Sarepta Therapeutics of Cambridge, Massachusetts, reports it has been notified by the Food and Drug Administration (FDA) that the agency was unable to complete its review of Sarepta’s New Drug Application (NDA) for the Duchenne muscular dystrophy (DMD) drug candidate eteplirsen by the Prescription Drug User Fee Act (PDUFA)…