Benitec Executive Chairman and Chief Executive Officer, Jerel A. Banks, M.D., Ph.D., will share an updated presentation detailing the significant, durable, improvements in dysphagic symptom burden and ...

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.

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 ...

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 ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...

Catalyst is offering patients and their caregivers a personalized program called Catalyst Pathways to provide guidance and financial support. Agamree ® (vamorolone) oral suspension has been made ...

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an ...

The MarketWatch News Department was not involved in the creation of this content. PARAMUS, N.J., March 9, 2026 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ...

HOUSTON – There’s a new type of therapy that can help kids with Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) affects one in 3,600 male births, and it can lead to immobility, and ...

William is a seven-year-old boy from Southern Oregon living with Duchenne muscular dystrophy. Now, he is preparing to undergo a surgery that has never been tried before, a procedure designed ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Eight therapies ...