Risdiplam-Treated Infants With Type 1 Spinal Muscular Atrophy
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The New England Journal of Medicine
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Risdiplam-Treated Infants With Type 1 Spinal Muscular Atrophy Versus Historical Controls
N. Engl. J. Med 2021 Jul 29;385(5)427-435, BT Darras, R Masson, M Mazurkiewicz-Bełdzińska, K Rose, H Xiong, E Zanoteli, G Baranello, C Bruno, D Vlodavets, Y Wang, M El-Khairi, M Gerber, K Gorni, O Khwaja, H Kletzl, RS Scalco, P Fontoura, L ServaisFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.