Tenaya’s Highly Selective HDAC6 Inhibitor TN-301 Outperformed Approved Pan-HDAC Inhibitor Givinostat in Improving Muscle Function and Correcting ...

Patients treated with low dose BB-301 and high dose BB-301 experienced significant improvements in throat closure, throat emptying, and total dysphagic symptom burden - OPMD Patients treated with low ...

Shares of Dyne Therapeutics gained after the company shared positive data from a continuing trial of a potential Duchenne muscular dystrophy treatment. The stock rose 14%, to $16.93, midday Monday.

ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced the presentation of ten abstracts at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Muscular dystrophies are a group of more than 30 genetic disorders characterized by progressive muscle weakness and wasting. These disorders are typically associated with defects in muscle ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).

Muscular dystrophy came for my vision first, although I did not know it at the time. On the second Friday morning of April 2020, I realized quite suddenly I couldn’t read out of my right eye. I could ...

Luke Criado, a 12-year-old from San Antonio, is battling Duchenne Muscular Dystrophy while staying active in power soccer.