Patricia Inácio, PhD, science writer —

Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type 2A (LGMD2A). LGMD2A, also known as calpainopathy, is the most common subtype of limb girdle muscular dystrophy (LGMD),…

PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which recognize the efforts of nonprofit organizations working to serve the Duchenne muscular dystrophy community. This year’s winners are patient organizations located in Canada, Argentina, Brazil, Croatia, Hungary, Ireland, Slovenia and Turkey.

A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will begin enrolling boys, ages 4 to 7, in the coming months, Catabasis Pharmaceuticals announced. Top-line results from this trial, to be called POLARIS, are expected in April 2020, and this…

Summit Therapeutics is stopping the clinical development of ezutromid, which it was investigating as a Duchenne muscular dystrophy (DMD) treatment, after a Phase 2 clinical trial testing the therapy failed to reach both its primary and secondary objectives. “These data come as a great disappointment to us and to…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, a dystrophin gene therapy aiming to treat Duchenne muscular dystrophy (DMD). FDA, in its letter, said that Solid Biosciences, the therapy’s developer, had addressed all questions of concern that led to…

The Centers for Disease Control and Prevention (CDC) released new, updated guidelines for the management of Duchenne muscular dystrophy (DMD) patients. Researchers highlighted the role of rehabilitation techniques and the importance of physiatrists to improve function and quality of life. The study,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD),its developer, Genea Biocells, announced. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA…

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against Exondys 51 (eteplirsen) as a therapy for Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51 of the dystrophin gene, the treatment’s developer, Sarepta Therapeutics, has announced. The company…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational therapy Sarconeos for Duchenne muscular dystrophy (DMD), Biophytis, the therapy’s developer, announced. The company also has filed a similar application with the European Medicines Agency (EMA). A decision is is expected…

Exondys 51 (eteplirsen), an RNA-targeted therapy marketed by Sarepta Therapeutics, is the first approved treatment targeting the underlying cause of Duchenne muscular dystrophy (DMD). Sarepta is continuing to refine its RNA-targeted therapies, with a goal of being able to transport more of the treatment…