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…
My experiences writing this column over the past year have been fabulous. But it was daunting at first to put pen to paper to capture what so many people feel. My first column talked about starting something new and the excitement this brings. But the reality is that the…
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…
Most people with a chronic illness inevitably learn to expect the unexpected with their health. No matter how meticulously I attempt to plan around my body’s needs, mitochondrial myopathy sometimes has a different agenda. I lose control over my body and feel as though I must bend to the…
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).
Stretching has been part of my daily life since I was diagnosed with Duchenne muscular dystrophy (DMD) about 17 years ago. Every night, my parents would reserve 30 minutes to do passive stretching, and I saw a physical therapist regularly. We didn’t do it because we wanted to, but…
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…
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