Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...
Regenxbio (NASDAQ:RGNX) and Solid Biosciences (NASDAQ:SLDB) witnessed contrasting fortunes on Tuesday after Sarepta ...
Belfast Live on MSN29m
'The doctors told me I would never walk but now they're shocked to see I can fly planes'Hero medics helped me regain my ability to swallow and get my life back' He told Belfast Live: "I was born with muscular ...
Ed Kaye, former CEO of Sarepta Therapeutics, is stepping down from his current role as chief executive of a Boston-area ...
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...
The FDA expanded the indication of eculizumab (Soliris) to include pediatric patients ages 6 and older with generalized ...
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...
Sarepta Therapeutics shares dropped 20% after a patient's death raises Elevidys safety concerns. Read why I am downgrading ...
Sarepta Therapeutics Inc.'s stock tumbled 23% on Tuesday after the biotech said a patient who used its gene therapy Elevidys for the rare disease Duchenne muscular dystrophy has died of acute liver ...
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
The Food and Drug Administration has accepted Capricor Therapeutics' application seeking approval of its cell therapy deramiocel for muscular dystrophy patients and granted it a priority review.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback