News

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…

A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle deterioration and results in muscle regeneration in animal models of Duchenne muscular dystrophy (DMD) without triggering an immune response, a new study reports. The study, “Non-immunogenic…

Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD) being developed by ReveraGen Biopharma, continues to improve muscle function in DMD patients with fewer side effects than standard corticosteroids, topline data in 23 boys in an ongoing Phase 2 trial show. These findings were detailed in a late-breaking…

To address the need in precision medicine for human biological specimens, CureDuchenne is creating a biobank to provide Duchenne muscular dystrophy (DMD) researchers with a collection of blood and skin tissue samples. The overarching goal is to use the biorepository to fuel and advance DMD research. The CureDuchenne Biobank…