Healthcare Costs in Commercially Insured Pediatric Patients with Generalized Myasthenia Gravis in the United States

Factors Associated with Recurrent Clinical Events among Pediatric Generalized Myasthenia Gravis Patients in the United States

Dystrophin Deficiency Alters Phospholipid and Sphingolipid Homeostasis in Human Cardiac Organoids

Treatment of SMA in a Preterm Infant with Elevated Anti-AAV9 Antibody Titers: A Case Report

RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Phase I/II Clinical Data