Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Author:

Darras Basil T.1,Masson Riccardo1,Mazurkiewicz-Bełdzińska Maria1,Rose Kristy1,Xiong Hui1,Zanoteli Edmar1,Baranello Giovanni1ORCID,Bruno Claudio1,Vlodavets Dmitry1,Wang Yi1,El-Khairi Muna1,Gerber Marianne1,Gorni Ksenija1,Khwaja Omar1,Kletzl Heidemarie1,Scalco Renata S.1,Fontoura Paulo1,Servais Laurent1

Affiliation:

1. From the Department of Neurology, Boston Children’s Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) — both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children’s Hospital...

Funder

F. Hoffmann-La Roche

Publisher

Massachusetts Medical Society

Subject

General Medicine

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