Marisa Wexler, MS, senior science writer —

The conditional approval of Translarna (ataluren) in Europe as a treatment for Duchenne muscular dystrophy (DMD) — in place for nearly a decade — should now not be renewed, a committee of the European Medicines Agency (EMA) has recommended. The EMA’s Committee for Medicinal Products for Human Use…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Avidity Biosciences. Orphan drug designation is given to therapies with the potential to treat rare disorders, those that affect fewer…

A new project hopes to improve data collection related to Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), with the aim of accelerating research for both diseases. The effort is a collaboration between the Duchenne Data Foundation (DDF) and the Duchenne Regulatory Science Consortium (D-RSC), a…

The first person with limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9) has been dosed with the gene therapy AB-1003 in a clinical trial, according to an announcement from the therapy’s developer Asklepios BioPharmaceutical (AskBio). “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is…

Synthetic biology company bit.bio has launched two new human muscle cell models to advance research into new treatment strategies for Duchenne muscular dystrophy (DMD). The two models, dubbed ioSkeletal Myocytes DMD Exon 44 Deletion, and ioSkeletal Myocytes DMD Exon 52 Deletion, are the eighth and ninth products that…

After a year of treatment with the experimental oral therapy EDG-5506 in a Phase 1 study, most men with Becker muscular dystrophy (BMD) show stable or improved motor function — a marked contrast to the disease’s natural course, where motor function worsens over time. One-year findings in the…

Catalyst Pharmaceuticals has entered a licensing agreement for the North American rights to vamorolone, a dissociative corticosteroid under review in the U.S. and Europe for Duchenne muscular dystrophy (DMD). Under the deal, Catalyst will give vamorolone’s developer Santhera Pharmaceuticals an upfront cash payment of $75 million,…

Three advocacy organizations are teaming up to run a clinical trial to test if efgartigimod, an approved treatment for certain autoimmune diseases, might let more people with Duchenne muscular dystrophy (DMD) benefit from gene therapies. CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD)…

Avidity Biosciences is planning to double the dosage of AOC 1001 being given to a dozen people with myotonic dystrophy type 1 (DM1) who are receiving the experimental therapy in the MARINA-OLE clinical trial. The U.S. Food and Drug Administration (FDA) had placed a partial clinical hold…

An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). While the FDA is not obligated to abide by the committee’s vote, the agency will consider…