Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
I can’t believe it’s been 13 years since I started exhibiting muscle loss. A lot has happened since then. My loss is slow but steady. Even when I plateau, I look back and realize that I’ve weakened over time. But if my column has one overarching theme, it’s that we…
Owing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another clinical hold on a Phase 1/2 clinical trial of SGT-001, an investigational gene therapy for Duchenne muscular dystrophy (DMD). Solid Biosciences, the company developing SGT-001, is working…
The clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with Duchenne muscular dystrophy (DMD) has been discontinued, according to a letter to the DMD community by Roche and Genentech. “We recognize this news is deeply disappointing for the Duchenne community, especially in…
Fall and winter have always been my favorite seasons. I eagerly await the cooler weather by prematurely washing my sweaters and drinking warm beverages. This year, I’m particularly excited for the changing of seasons because of some maddening mosquitoes. This summer, a non-native mosquito species terrorized me and other…
The Muscular Dystrophy Association’s (MDA) efforts to support and advocate for a wide range of neuromuscular diseases are felt by patient communities nationwide. While the MDA’s scope is indeed a national one, the organization reaches patients and families in a variety of different ways, including local events such as the…
Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with Duchenne muscular dystrophy (DMD), a study has found. This cell therapy, by Dystrogen Therapeutics, will be tested in a clinical trial involving DMD patients,…
The investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root cause of muscle degeneration in facioscapulohumeral muscular dystrophy (FSHD), new research in patient-derived cells and in a mouse model suggests. This supports a way forward in developing the first…
It can sometimes feel like everything is against us when doctor appointments and physical barriers bombard us. If we focus on the day-to-day challenges, we forget what we’re doing on earth. I recently discovered that practical goal setting helps you accomplish more than you ever thought possible. The mere act…
An experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne muscular dystrophy (DMD) in animal models of the disease, a study shows. These findings suggest BIIB100’s potential for people with DMD, which the researchers hope will be evaluated in a future Phase 1 trial.
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