Forest Ray PhD,  —

The French National Agency for Medicines and Health Products Safety has cleared Généthon to begin a clinical trial testing the safety and efficacy of GNT0004, a potential gene therapy for Duchenne muscular dystrophy (DMD), in France. “From translational research to clinical application, Généthon has supported this candidate drug,…

A new collaboration between Eli Lilly and Precision BioSciences aims to develop gene editing therapies for genetic disorders, initially focusing on Duchenne muscular dystrophy (DMD) and two other undisclosed targets, the two companies announced. Precision licensed its ARCUS genome editing platform to Lilly and will lead…

Note: This story was updated Dec. 4, 2020, to note that Teji Singh’s title is executive medical director of Sarepta, rather than the chief medical officer.  Sarepta Therapeutics is about to launch a Phase 1 trial of SRP-9001, its investigational gene therapy for Duchenne muscular dystrophy (DMD),…

The Myotonic Dystrophy Clinical Research Network (DMCRN) is asking adults with myotonic dystrophy type 1 (DM1) to participate in a large natural history study aimed at improving clinical trials by better understanding the full spectrum of the disorder. “Prior drug studies have been limited by a lack…

The Muscular Dystrophy Association (MDA) and DNAnexus have introduced a digital platform to improve patient care and accelerate treatment discovery for muscular dystrophy and other neuromuscular disorders. The neuroMuscular ObserVational Research (MOVR) Visualization and Reporting Platform (VRP) provides easy-to-use tools for in-depth analysis of large neuromuscular patient…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease status to AMO Pharma‘s investigational therapy AMO-02 (tideglusib), now entering clinical testing in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), also known as Steinert disease. “This Rare Pediatric Disease designation highlights the significant…

Italfarmaco has now completed recruitment — delayed in the spring due to the COVID-19 pandemic — of boys with Duchenne muscular dystrophy (DMD) for its ongoing Phase 3 trial of givinostat, according to a top company officer. In another milestone, Givinostat has “just received” rare pediatric disease…

September is Duchenne Action Month, an awareness, fund-raising, and educational campaign supporting people with Duchenne muscular dystrophy (DMD) Hosted by Parent Project Muscular Dystrophy (PPMD), each week of the month is organized around a different theme, with scheduled events, as well as various resources and social media…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. The FDA conditionally approved Viltepso earlier this month, and continued approval remains contingent upon confirmation of clinical benefit in the Phase 3 RACER53…