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Parent Project Muscular Dystrophy (PPMD) will use PerkinElmer‘s recently federally authorized test to aid newborn screening for Duchenne muscular dystrophy (DMD) in a newly launched pilot program in New York state. Results from the two-year Newborn Screening Pilot for Duchenne Muscular Dystrophy, launched in collaboration with consortia…

Note: This story was updated Dec. 17, 2020, to note that preclinical studies testing this form of gene therapy were conducted in animals that naturally develop DMD, not genetically engineered animals. Genethon and Sarepta Therapeutics have extended their original agreement to develop GNT0004, a potential …

AMO Pharma has begun enrollment for a Phase 2/3 trial that will assess the experimental oral therapy AMO-02 (tideglusib) in children and adolescents with congenital myotonic dystrophy type 1 (DM1). The randomized, double-blind trial (NCT03692312) is intended to support regulatory approval of AMO-02 for congenital…

Rich Horgan’s younger brother, Terry Horgan, is the inspiration behind Cure Rare Disease, a Boston-based nonprofit Rich established in 2017 to help scientists develop personalized therapies for people with rare diseases. Terry, 24, has Duchenne muscular dystrophy (DMD), which is the organization’s first focus. Besides Terry, three other…

Duchenne UK is partnering with Catabasis Pharmaceuticals to provide financial support for a Phase 2 clinical trial testing the experimental oral therapy edasalonexent in non-ambulatory boys and men with Duchenne muscular dystrophy (DMD). The charity is granting more than $600,000…

Montana, the fourth-largest U.S. state in area, also is one of the least-populated — with maybe half a dozen Duchenne muscular dystrophy patients among its 1.1 million inhabitants. One of them is 5-year-old Grant Heaton, the son of cattle ranchers Paul and Laura Heaton. Diagnosed with Duchenne at age…

Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on advancing the development of SGT-001, its gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD). SGT-001 uses a viral vector to deliver a…

Tonya Dreher wanted to do something dramatic on behalf of Hope for Gus, the New Hampshire-based foundation inspired by her son, Gus, 14, who has Duchenne muscular dystrophy (DMD). In 2015, frustrated with her fundraising efforts on behalf of DMD research, she came up with the idea of…

Parent Project Muscular Dystrophy (PPMD) has granted $465,000 to Renzhi Han, PhD, a professor at the Ohio State University College of Medicine, to advance research into a targeted gene editing approach that may protect cardiac and skeletal muscle function in people with Duchenne muscular dystrophy (DMD).

Astellas Pharma recently agreed to acquire Audentes Therapeutics, a move it expects will result in faster development of potentially best-in-class therapies for rare neuromuscular diseases, including muscular dystrophy (MD). Audentes’ vectorized exon-skipping technology — which uses a modified adeno-associated virus (AAV) vector to allow cells to…