Sarepta Therapeutics said a patient taking its treatment for Duchenne muscular dystrophy has died from acute liver failure.
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
Acute liver failure (ALF) is a rare but devastating syndrome. ALF in children differs from that observed in adults in both the etiologic spectrum and the clinical picture. Specific therapy to ...
The company said that more than 800 people have been treated with Elevidys, with only one experiencing liver failure.