Finkel, R. S., Hughes, S. H., Parker, J., Civitello, M., Lavado, A., Mefford, H. C., Mueller, L., Kletzl, H. (2025). Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy. New England Journal of Medicine. https://doi.org/10.1056/nejmc2300802