BioMarin returns to FDA with a new pitch for hemophilia gene therapy — and a promise to get to the bottom of durability
Two years ago, the FDA slapped a surprise rejection on BioMarin’s BLA for its gene therapy for hemophilia A, demanding two-year data from a …
Sign up to read this article for free.
Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.
