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…
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…
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…
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.
Parent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at Nationwide Children’s Hospital and the University of Missouri to help better understand and improve the treatment of cardiac disease in people with Duchenne muscular dystrophy (DMD). The funding falls under PPMD’s…
Treatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne muscular dystrophy (DMD), real-world data show. The most recent results from the ongoing STRIDE registry — an observational study across more than 50 care centers in Europe and Israel — were presented at…
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s vamorolone for the treatment of Duchenne muscular dystrophy (DMD). PIM designation indicates that the MHRA considers vamorolone likely to offer major therapeutic advantages for DMD patients. The MHRA will next…
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