Pediatric Neuromuscular Diseases: From Myasthenia to Gene Therapy - Alex Fay, MD, PhD (1/22/26)

Title: Pediatric Neuromuscular Diseases: From Myasthenia to Gene Therapy and Back Speaker: Alex Fay, MD, PhD Associate Professor of Neurology UCSF Department of Neurology How does the treatment of classic autoimmune diseases inform the management of complications from modern gene therapy? Gain insights on handling the toxicity of gene therapy with Alex Fay, MD, PhD, associate professor of Neurology at UCSF. A UCSF alumnus with a doctorate in biophysics, Dr. Fay combines a deep understanding of molecular mechanisms with clinical expertise in neuromuscular diseases including muscular dystrophy and spinal muscular atrophy. His work focuses on bringing targeted therapeutics to children with rare neurogenetic diseases. In this presentation, Dr. Fay explains the mechanisms of emerging gene therapies for pediatric neurological diseases and the diagnostic challenges of pediatric myasthenia gravis. Crucially, he discusses how established strategies for treating autoimmune disease are now providing the blueprint for managing the toxicity and complications of gene therapy. Learning Objectives: •To understand diagnostic and treatment challenges in pediatric myasthenia gravis. •To understand the mechanisms, efficacy, and toxicity of gene therapy across pediatric neurological diseases. •To understand how the treatment of autoimmune diseases like myasthenia gravis informs the management of complications of gene therapy.