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 ...

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 ...

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 ...

Consuming more dietary protein may benefit people with various types of muscular dystrophy, according to a recent study.

NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today ...

Shares of Precision BioSciences (NASDAQ:DTIL) rose 13% on Monday after the U.S. Food and Drug Administration granted Fast Track designation to PBGENE-DMD, the company’s gene-editing therapy for ...

Comparable long-term effectiveness to classic corticosteroids, both prednisone (p=0.8587) and deflazacort (p=0.6544), based on time to loss of ...

Dyne announced the results of new analyses of cardiac and pulmonary function amongst all DELIVER participants who were randomized to z-rostudirsen treatment at baseline (any dose 1) and for whom ...

New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history - - Data expand on previously reported results demonstrating that z-ros ...

Keros Therapeutics, Inc. ('Keros” or the 'Company”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on ...

Santhera Pharmaceuticals is leveraging fresh clinical evidence to strengthen the market position of its flagship therapy, Agamree. The company recently presente ...