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Sarepta Therapeutics has been hit with an FDA clinical hold on its trial of SRP-5051 (vesleteplirsen) for the treatment of Duchenne muscular dystrophy after a trial participant experienced a serious adverse event (SAE) that could have been life-threatening. Read More
At long last, PTC Therapeutics has announced positive results from a study of Translarna (ataluren), the company’s drug for patients with nonsense mutation Duchenne muscular dystrophy. Read More
Roche is finally throwing in the towel on crenezumab, the antiamyloid antibody researchers hoped might prevent the disease in a group of South American families genetically destined to develop it at a very young age. Read More
Acadia is having another go at Nuplazid (pimavanserin), hoping to convince the FDA’s Psychopharmacologic Drugs Advisory Committee that the antipsychotic should win a new indication as a treatment for dementia-related hallucinations and delusions. Read More
Due to a very low rate of hospitalization or death observed in the standard-risk patient population, Pfizer has decided to end enrollment in the study. Read More
Vertex Pharmaceuticals’ and CRISPR Therapeutics reported positive follow-up data from two late-stage clinical trials showing continued benefit from using their gene-editing treatment for people with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD). Read More