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. 2025 Mar 13;392(11):1138-1140.

doi: 10.1056/NEJMc2300802. Epub 2025 Feb 19.

Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy

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, Affiliations

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

Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy

Richard S Finkel et al. N Engl J Med. 2025.

. 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

PubMed Disclaimer

Comment in

Further Comments on Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
Wadman RI, Groen EJN, van der Pol WL. Wadman RI, et al. N Engl J Med. 2025 Jul 3;393(1):103. doi: 10.1056/NEJMc2504911. N Engl J Med. 2025. PMID: 40601958 No abstract available.
Further Comments on Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
Yeo CJJ, De Vivo DC, Darras BT. Yeo CJJ, et al. N Engl J Med. 2025 Jul 3;393(1):103-104. doi: 10.1056/NEJMc2504911. N Engl J Med. 2025. PMID: 40601959 No abstract available.
Further Comments on Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy. Reply.
Finkel RS, Mueller L, Kletzl H. Finkel RS, et al. N Engl J Med. 2025 Jul 3;393(1):104. doi: 10.1056/NEJMc2504911. N Engl J Med. 2025. PMID: 40601960 No abstract available.

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