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 ...

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 ...

– Company to host an Industry Forum to discuss the lived experience of Becker and clinical advancements featuring a leading neuromuscular disease expert and a patient advocate – Edgewise is sponsoring ...

"Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We ...

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 ...

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 ...

New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history -- Data expand ...

A research team led by Associate Professor Hidetoshi Sakurai and Researcher Nana Takenaka-Ninagawa recently demonstrated the superior therapeutic potential of iPS cell-derived mesenchymal stromal ...

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 ...