News

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…

Experimental therapy ATL1102 improved upper limb strength and function in six boys with Duchenne muscular dystrophy (DMD) unable to walk, according to early results of an ongoing Phase 2 clinical trial. These results also indicate that the therapy has been well-tolerated with no safety concerns reported so far.

Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of a gene therapy to prevent heart failure linked to Duchenne (DMD) and Becker muscular dystrophy (BMD), and another to create better measures of treatment responses in DMD clinical trials. PPMD, a nonprofit organization…

Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes limb-girdle muscular dystrophy (LGMD) type 2B. Edasalonexent, formerly CAT-1004, is a small molecule designed…

Dyne Therapeutics announced that it has joined in supporting an ongoing global and observational natural history study to better inform the development of treatments for facioscapulohumeral muscular dystrophy (FSHD). The company’s support — the amount was not disclosed — will help to bring new sites across Europe into…

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of a biomarker of muscle damage in three children with limb girdle muscular dystrophy (LGMD) type 2E. The trial (…

Nippon Shinyaku filed a request for the approval of viltolarsen (NS-065/NCNP-01) to treat Duchenne muscular dystrophy amenable to exon 53 skipping with the U.S. Food and Drug Administration (FDA), the company announced in a press release. The request came in the form of a rolling new drug application (NDA) whose…

Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular dystrophy (DMD) within the organization’s pilot newborn screening program in New York. The $5 million pilot program, launched in collaboration with the American College of Medical Genetics and Genomics (ACMG) and New York State,…