New research into a mechanism underlying Duchenne muscular dystrophy (DMD) — which revealed a chemical modification in gene activity — has shed light on potential treatments for the muscle-wasting disease, and may aid in diagnosis, according to scientists. DMD, the study showed, is characterized by a specific pattern of…
Satellos Bioscience has designated SAT-3153 as a potential muscle regeneration treatment for Duchenne muscular dystrophy (DMD). The therapeutic candidate now will be tested in pre-investigational new drug (pre-IND) enabling studies intended to provide evidence to obtain regulatory approval for conducting a clinical trial. “We are thrilled to be in…
Santhera Pharmaceuticals has asked the European Medicines Agency (EMA) to approve vamorolone to treat Duchenne muscular dystrophy (DMD). The agency’s assessments of new approval requests — in the form of marketing authorization applications — take up to 210 days, but can be longer if the Committee for Medicinal Products…
The Muscular Dystrophy Association (MDA) has awarded more than $17 million to projects aimed at advancing research and developing new treatments in muscular dystrophy (MD) and other neuromuscular diseases. The funding adds to the more than $1 billion the MDA has already invested in research. “The Muscular Dystrophy…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
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