The stock of small-cap biotech Wave Life Sciences Inc. soared 51% Tuesday after the company reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne ...

Mesoblast (MESO) added ~6% in the premarket on Wednesday after the FDA cleared its Investigational New Drug application that sought approval to conduct a study to test its cell therapy, Ryoncil, in ...

When a Harford County mom noticed her son was moving slower than his siblings, she became concerned.After years of trying to find an answer, she got one that she wasn't prepared for.Davia Jones said ...

Eight years ago, President Donald Trump signed the Right to Try legislation to give people with terminal illnesses a chance at life. Now his Food and Drug Administration has undermined those ...

March 11 (Reuters) - Regenxbio (RGNX.O), opens new tab said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD). But the company’s efforts to expand its use to a new indication is coming ...

With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. Late Thursday, PTC revealed that it ...

Sarepta shares rise more than 12% Analysts see potential for improved perception, commercial uptake Elevidys shows up to 73% reduction in rate of functional decline Jan 26 (Reuters) - Sarepta ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

Sarepta Therapeutics (SRPT) shares fell after its late-stage trial for two gene therapies, AMONDYS 45 (casimersen) and VYONDYS 5 (golodirsen), aimed at treating Duchenne muscular dystrophy, did not ...

People with Duchenne muscular dystrophy have a shorter-than-average life expectancy due to changes in muscles that affect breathing, heart activity, and other functions. However, treatment advances ...