Accelerating therapeutic development and clinical trial readiness for STXBP1 and SYNGAP1 disorders

Nicolas Marotta, Michael J. Boland, Benjamin L. Prosser

2024Current problems in pediatric and adolescent health care15 citationsDOI

Topics & Concepts

Clinical trialMedicinePsychiatryPathologyCellular transport and secretionGenetics and Neurodevelopmental DisordersLysosomal Storage Disorders Research