Hemophilia A

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) valoctocogene roxaparvovec investigational gene therapy for the treatment of adults with severe hemophilia A.  RMAT is an expedited...

Baird has initiated coverage on Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) with an Outperform rating, citing diversified pipeline with multiple near-term catalysts. It also upgraded BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to Outperform with a $110 price target. Ultragenyx Ultragenyx's...