St. Jude Children’s Research Hospital and University College London reported successful findings upon completing a follow-up of patients who received gene therapy for hemophilia B, according to a release.
The researchers said the treatment was “safe and effective long-term” after observing a group of patients for 13 years post gene transfer. The 10 adults “have all maintained a steady level of factor IX and an excellent benefit in terms of freedom from bleeding.” Yearly bleeding decreased nearly tenfold, according to St, Jude.
Principal investigator of the study, Ulrike Reiss, MD, reported no side effects or toxic events.
