The National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council has issued two new documents on November 21, 2018 that were approved by NHF’s Board of Directors on December 6, 2018. The first document addresses the risks associated with gene therapy clinical trials. The second document provides recommendations regarding the use of emicizumab-kxwh (Hemlibra®) for patients with and without inhibitors.
MASAC Document # 254
MASAC DOCUMENT REGARDING RISKS OF GENE THERAPY TRIALS FOR HEMOPHILIA
MASAC Document #258
