News

PTC Therapeutics, Inc. announced Wednesday that it has received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) with regard to its New Drug Application (NDA) for ataluren (trade name, Translarna), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy…

One hundred and nine members of the U.S. House of Representatives signed a letter to the U.S. Food and Drug Administration (FDA) calling for the agency to immediately increase efforts to mobilize all available tools, resources, and authorities to advance new and more effective treatments for Duchenne muscular dystrophy. The bipartisan appeal…

Tarix Orphan LLC recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127, the company’s lead candidate for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD). Early-onset LAMA2-related muscular dystrophy (MD) occurs in nearly one in every 30,000 people and is responsible for…

Pfizer, in collaboration with Newcastle University, reports finding four serum biomarkers for three types of muscular dystrophy. The markers may be valuable for monitoring disease progression and treatment response in both preclinical and clinical studies. Researchers analyzed previously collected serum samples from three types of muscular dystrophies — 38 Becker muscular dystrophy (BMD) and…

UCLA scientists have developed a potential gene therapy approach for Duchenne muscular dystrophy (DMD) using CRISPR/Cas9 technology and stem cells. If the new treatment proves successful and reaches the clinic — possibly in the next decade — it could be applied to 60 percent of all Duchenne patients. CRISPR/Cas9 is a gene-editing technology that allows targeting and…

A discovery clears up the confusion about the molecular factors involved in muscle repair. The research, published in the journal e-life, shows that a protein complex containing the factors TBP and TFIID is essential to activate genes responsible for adult muscle tissue regeneration. Adult muscles contain stem cells that can be activated…

Catabasis Pharmaceuticals announced it will present data on CAT-1004 at the XIV International Conference on Duchenne and Becker Muscular Dystrophy to be held in Rome, Italy, on Feb. 13-14. Joanne Donovan, chief medical officer of Catabasis, will talk about the drug, now in clinical testing, and the design of the MoveDMD…

Sarepta Therapeutics, a developer of RNA-targeted treatments, announced that the U.S. Food and Drug Administration (FDA) is extending by another three months its review of the company’s New Drug Application (NDA) for eteplirsen, a potential treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A reason for the extension was…

Catabasis Pharmaceuticals and the Muscular Dystrophy Association (MDA) have announced a partnership that will support Part B of the MoveDMD clinical trial, currently evaluating the effectiveness and safety of Duchenne muscular dystrophy (DMD) treatment candidate CAT-1004 in children. CAT-1004, believed to be a disease modulator regardless of mutation, is…

In recognition that people with muscular dystrophy are living longer than even recently thought possible, the Muscular Dystrophy Association (MDA) has named a young man as its latest National Goodwill Ambassador — marking the first time in the program’s 64-year history that a child hasn’t been selected for that role.