A hemophilia patient hero at zero, BioMarin to seek FDA OK for gene therapy. But will the effect last?

A potential one-shot-and-you're-done hemophilia A treatment from BioMarin Pharmaceutical Inc. will target regulatory approval in the United States and Europe after showing dramatic decreases in bleeding during clinical trials. But in a competitive market to find the next big and expensive treatment to help — or even cure — hemophilia A patients, questions linger around how long the treatment sticks with patients. BioMarin (NASDAQ: BMRN) apparently didn't allay those concerns Tuesday: The San…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news