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Sarepta reports second patient death after treatment with Duchenne gene therapy
Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who received the treatment.
Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...
Supernus Pharmaceuticals said it is acquiring Sage Therapeutics, the distressed maker of a drug for postpartum depression, ...
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...
Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...
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Cardiac Complications of Duchenne Muscular DystrophyCardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies ...
The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...
Sarepta Therapeutics shares dived 42% in premarket trading after the company disclosed a second reported case of acute liver failure that resulted in a death tied to its Duchenne muscular dystrophy ...
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