In a decision that surprised many, the U.S. Food and Drug Administration (FDA) last week denied approval of Biomarin’s FVIII gene therapy, branded Roctavian. The...
Dr. Steven Pipe, Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program in Ann Arbor Michigan, presented an update at the World Federation of...
The global bleeding disorder community received an advance look at new international guidelines for the management of hemophilia at the WFH Virtual Summit 2020, at...
WFH Gene Therapy Registry, presented by Dr. Barbara Konkle For many years gene therapy has been in the future – but now, for an increasing...
The CHS recently learned of two deaths of children under five years old in a clinical trial using AAV8-based vectors in another rare disease area,...
