SAN FRANCISCO — “No one ever got really rich shorting stocks,” said Joe Edelman, the founder and CEO of Perceptive Advisors, a $7.5 billion biotech
Confounding data from a Pfizer clinical trial has rattled the field of gene therapy for Duchenne muscular dystrophy, raising more questions about the regulatory standard
To hear Sarepta CEO Doug Ingram tell it, the results Monday afternoon from a large trial of its gene therapy for Duchenne muscular dystrophy marked
Sarepta Therapeutics said Monday afternoon that its gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large
The Food and Drug Administration granted conditional approval Thursday to the first gene therapy for Duchenne muscular dystrophy. Regulators restricted the treatment to younger patients,