Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.

Richard S Finkel, Samuel H Hughes, JulieAnn Parker,Matthew Civitello, Alfonso Lavado,Heather C Mefford, Lutz Mueller,Heidemarie Kletzl, Prenatal SMA Risdiplam Study Group

The New England journal of medicine（2025）

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