Advancing Care of a Devastating Disease Through Gene Therapy
University of California, San Francisco (UCSF)A new international trial is investigating the efficacy and safety of gene therapy in patients with Hurler syndrome and will compare results of gene therapy against BMT which is the current standard of care. UCSF Benioff Children’s Hospitals are among three sites in the U.S. and seven in the world now enrolling patients for the clinical trial.