Q&A About the Use of Gene Transfer Therapy for the Treatment of Rare Pediatric Neurologic Disorders

Pediatric neurologist Tom Crawford discusses how gene transfer therapy is being used to treat rare neurologic diseases in children, how it’s used at Johns Hopkins to treat infants diagnosed with spinal muscular atrophy (SMA), and challenges the field of gene transfer therapy must address in the near future.

Created by

Johns Hopkins Medicine

Related Presenters

Thomas Crawford, MD

Thomas Crawford, MD

Co-Director, Muscular Dystrophy Association Clinic

View full profile