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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.
Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...
Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...
Supernus Pharmaceuticals said it is acquiring Sage Therapeutics, the distressed maker of a drug for postpartum depression, ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...
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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, ...
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