Litcius/Paper detail

Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy

Richard S. Finkel, Samuel H. Hughes, J. W. Parker, Matthew Civitello, Alfonso Lavado, Heather C. Mefford, Lutz Mueller, Heidemarie Kletzl

2025New England Journal of Medicine53 citationsDOI

Topics & Concepts

Spinal muscular atrophyMedicineAtrophyPhysical medicine and rehabilitationPathologyDiseaseNeurogenetic and Muscular Disorders ResearchCongenital Anomalies and Fetal SurgeryCardiac Structural Anomalies and Repair