Diana Bharucha-Goebal, MD, PhD

Children’s National Hospital
Washington, DC, and the National Institutes of Health (NIH)

Dr. Diana Bharucha-Goebel is Director of the Neuromuscular Program and Associate Professor at Children’s National Hospital (Neurology) and a Research Collaborator at the NIH (NINDS). She works on the evaluation of disease-focused outcome measures and trial design for neuromuscular diseases and studies the impact of novel therapies on care needs and emerging phenotypes for pediatric neuromuscular disorders. Following work co-leading a first-in-human intrathecal AAV-mediated gene transfer trial for giant axonal neuropathy (GAN), she also focuses on investigating how vector genome dose, route and immunologic considerations may impact the safety of viral mediated gene transfer targeting the peripheral nervous system.