Why four years is important for BioMarin, hemophilia patients

An experimental gene therapy for hemophilia A from BioMarin Pharmaceutical Inc. — promising a one-shot fix for the genetic cause of the bleeding disorder — continues to hold patients' bleeds to less than one a year after four years. The results of the ongoing study are important as San Rafael-based BioMarin's (NASDAQ: BMRN) gene therapy, called Roctavian and known scienti fically as valoctogene roxaparvovec, nears an Aug. 21 decision date from the Food and Drug Administration. If approved, it…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news