Children’s Hospital of Philadelphia Researchers Find Gene Therapy Shows Long-Term Benefits for Hemophilia B Patients
Apr 17, 2025
Researchers at Children’s Hospital of Philadelphia (CHOP) announced the results of the longest follow-up study to date for individuals receiving the gene therapy fidanacogene elaparvovec to treat hemophilia B, a rare, X-linked disorder resulting from a deficiency in coagulation factor IX (FIX) activity.