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Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study.
Cances, Claude; Vlodavets, Dmitry; Comi, Giacomo Pietro; Masson, Riccardo; Mazurkiewicz-Beldzinska, Maria; Saito, Kayoko; Zanoteli, Edmar; Dodman, Angela; El-Khairi, Muna; Gorni, Ksenija; Gravestock, Isaac; Hoffart, Janine; Scalco, Renata S; Darras, Basil T.
Affiliation
  • Cances C; AOC (Atlantic-Oceania-Caribbean) Reference Centre for Neuromuscular Disorders, Paediatric Clinical Research Unit/Paediatric Multi-Thematic Module CIC 1436, Neuropaediatric Department, Toulouse University Hospital, Toulouse, France. cances.c@chu-toulouse.fr.
  • Vlodavets D; Pediatric Clinical Research Unit, Pediatric Plurithematic Module, CIC 1436, Toulouse, France. cances.c@chu-toulouse.fr.
  • Comi GP; Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University, Moscow, Russia.
  • Masson R; Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy.
  • Mazurkiewicz-Beldzinska M; IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Neuromuscular and Rare Diseases Unit, Milan, Italy.
  • Saito K; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Zanoteli E; Department of Developmental Neurology, Medical University of Gdansk, Gdansk, Poland.
  • Dodman A; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
  • El-Khairi M; Department of Neurology, Faculdade de Medicina, Universidade de São Paulo (FMUSP), São Paulo, Brazil.
  • Gorni K; Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Gravestock I; Roche Products Ltd, Welwyn Garden City, UK.
  • Hoffart J; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Scalco RS; Personalized Healthcare Analytics, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Darras BT; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Orphanet J Rare Dis ; 17(1): 300, 2022 07 29.
Article in En | MEDLINE | ID: mdl-35906608
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Full text: 1 Collection: 01-internacional Health context: 2_ODS3 Database: MEDLINE Main subject: Muscular Atrophy, Spinal / Spinal Muscular Atrophies of Childhood Type of study: Observational_studies Limits: Humans / Infant Country/Region as subject: Asia / Europa Language: En Journal: Orphanet J Rare Dis Year: 2022 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Health context: 2_ODS3 Database: MEDLINE Main subject: Muscular Atrophy, Spinal / Spinal Muscular Atrophies of Childhood Type of study: Observational_studies Limits: Humans / Infant Country/Region as subject: Asia / Europa Language: En Journal: Orphanet J Rare Dis Year: 2022 Document type: Article