The National Hemophilia Foundation’s (NHF's) Medical and Scientific Advisory Council (MASAC) has issued a new document, which was adopted by NHF’s Board of Directors on August 13, 2019:
MASAC Document #256 emphasizes the clinical and scientific utility of liver biopsies in patients who receive liver-directed gene therapy via adeno-associated viral (AAV) vectors. The document includes a series of unanswered questions relevant to AAV biology, its integration into the human liver and subsequent factor expression. Answers to these questions would benefit both patients, investigators and the greater scientific community.
MASAC posits that many of these answers can be reached by incorporating serial liver biopsies into at least a subset of patients enrolled in AAV-based gene therapy clinical trials. To learn more, view and download MASAC Recommendation for Liver Biopsies in Gene Therapy Trials for Hemophilia.
View the complete list of current MASAC documents. To request a hard copy of any current documents, please contact HANDI, NHF’s information resource center: firstname.lastname@example.org or 1.800.424.2634.