Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls,The New England Journal of Medicine - X-MOL

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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2021-07-29 , DOI: 10.1056/nejmoa2102047
Basil T Darras ₁ , Riccardo Masson ₁ , Maria Mazurkiewicz-Bełdzińska ₁ , Kristy Rose ₁ , Hui Xiong ₁ , Edmar Zanoteli ₁ , Giovanni Baranello ₁ , Claudio Bruno ₁ , Dmitry Vlodavets ₁ , Yi Wang ₁ , Muna El-Khairi ₁ , Marianne Gerber ₁ , Ksenija Gorni ₁ , Omar Khwaja ₁ , Heidemarie Kletzl ₁ , Renata S Scalco ₁ , Paulo Fontoura ₁ , Laurent Servais ₁ ,

Affiliation

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 at Westmead and the University of Sydney, Sydney (K.R.); the Department of Pediatrics, Peking University First Hospital, Beijing (H.X.), and Children's Hospital of Fudan University, Shanghai (Y.W.) - both in China; the Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo (E.Z.); the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, the Department of Paediatrics, University of Oxford, Oxford (L.S.) - all in the United Kingdom; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute, Pirogov Russian National Research Medical University, Moscow (D.V.); Pharma Development, Safety (M.G.), Product Development Medical Affairs - Neuroscience and Rare Disease (K.G., P.F.), and Pharma Development Neurology (R.S.S.), F. Hoffmann-La Roche, and Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel (O.K., H.K.) - both in Basel, Switzerland; the Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, the Department of Pediatrics, University Hospital Liege, University of Liege, Liege, Belgium (L.S.); and I-Motion, Institut de Myologie, Assistance Publique Hôpitaux de Paris, Hôpital Armand Trousseau, Paris (L.S.).

New England Journal of Medicine, Volume 385, Issue 5, Page 427-435, July 2021.

中文翻译：

Risdiplam 治疗的 1 型脊髓性肌萎缩症婴儿与历史对照

新英格兰医学杂志，第 385 卷，第 5 期，第 427-435 页，2021 年 7 月。

更新日期：2021-07-29

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