Adeno-associated virus (AAV)–mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single ...

Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a ...

People with severe hemophilia A are at risk for prolonged bleeding events that can cause serious complications. To prevent these bleeding events, most people with this condition get injections of a ...

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) announced positive phase 3 gene therapy trial results in adults with Severe Hemophilia A. The study met all primary and secondary efficacy endpoints in ...

Hemophilia A is a rare, inherited condition that prevents your blood from clotting the way it should. People with hemophilia A do not make enough of a protein called factor VIII, which normally works ...

SAN RAFAEL, Calif., Jan. 2, 2020 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the New England Journal of Medicine (NEJM) published an independently peer-reviewed ...

A company developing a gene therapy for hemophilia plans to meet with US and European regulators to discuss potential filings for approval later this year after releasing interim Phase III data. San ...

A single infusion of the gene therapy valoctocogene roxaparvovec led to significantly reduced bleeding rates that persisted in men with severe hemophilia A, 2-year results from the phase III GENEr8-1 ...

SAN RAFAEL, Calif., June 17, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today additional data from its previously reported four-year update of an open-label Phase 1/2 ...

The latest trial results for its hemophilia A gene therapy suggest it may eliminate or significantly reduce the need for weekly infusions. By Todd Campbell – May 28, 2019 at 7:46PM EST Hemophilia A ...