Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy

N Engl J Med. 2025 Mar 13;392(11):1138-1140. doi: 10.1056/NEJMc2300802. Epub 2025 Feb 19.

Authors

Richard S Finkel¹, Samuel H Hughes¹, JulieAnn Parker², Matthew Civitello¹, Alfonso Lavado¹, Heather C Mefford¹, Lutz Mueller³, Heidemarie Kletzl³; Prenatal SMA Risdiplam Study Group

Collaborators

Prenatal SMA Risdiplam Study Group:
Richard S Finkel, Samuel H Hughes, JulieAnn Parker, Matthew Civitello, Alfonso Lavado, Heather C Mefford, Lutz Mueller, Heidemarie Kletzl, Elizabeth Fox, Liza-Marie Johnson, Chelsea G Goodenough, Jennifer L Harman, Gang Wu, Soham Sengupta, Emily S Bonkowski, Soniya N Pinto, Mary Ellen Hoehn, John H Bartley, Gaurav Seth, Kathryn R Wagner

Affiliations

¹ St. Jude Children's Research Hospital, Memphis, TN.
² OBGYN Partners of Augusta, Augusta, GA.
³ F. Hoffmann-La Roche, Basel, Switzerland.

PMID: 39970420
DOI: 10.1056/NEJMc2300802

No abstract available

Publication types

Letter