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Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.
Cornell, Nikki; Childs, Anne-Marie; Wraige, Elizabeth; Munot, Pinki; Ambegaonkar, Gautam; Chow, Gabriel; Hughes, Imelda; Illingworth, Marjorie; Majumdar, Anirban; Marini-Bettolo, Chiara; Parasuraman, Deepak; Spinty, Stefan; Willis, Tracey; Scoto, Mariacristina; Baranello, Giovanni.
Affiliation
  • Cornell N; The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute o
  • Childs AM; Leeds Royal Infirmary, Leeds, UK.
  • Wraige E; Evelina London Childrens Hospital, London, UK.
  • Munot P; The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute o
  • Ambegaonkar G; Addenbrookes Hospital, Cambridge, UK.
  • Chow G; Queen's Medical Centre Nottingham, Nottingham, UK.
  • Hughes I; Royal Manchester Children's Hospital, Manchester, UK.
  • Illingworth M; University Hospital Southampton, Southampton, UK.
  • Majumdar A; Bristol Royal Hospital for Children, Bristol, UK.
  • Marini-Bettolo C; John Walton Muscular Research Centre, Newcastle University and Newcastle upon Tyne Hospitals NHS Foundation trust.
  • Parasuraman D; University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
  • Spinty S; Alder Hey Children's Hospital, Liverpool, UK.
  • Willis T; The Robert Jones and Agnes Hunt Orthopaedic Hospital, Oswestry, UK.
  • Scoto M; The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute o
  • Baranello G; The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute o
J Neuromuscul Dis ; 11(2): 361-368, 2024.
Article in En | MEDLINE | ID: mdl-38189761
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Azo Compounds / Muscular Atrophy, Spinal / Spinal Muscular Atrophies of Childhood Type of study: Prognostic_studies Limits: Adolescent / Child / Child, preschool / Humans / Infant / Newborn Country/Region as subject: Europa Language: En Journal: J Neuromuscul Dis Year: 2024 Document type: Article Country of publication: Países Bajos
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Azo Compounds / Muscular Atrophy, Spinal / Spinal Muscular Atrophies of Childhood Type of study: Prognostic_studies Limits: Adolescent / Child / Child, preschool / Humans / Infant / Newborn Country/Region as subject: Europa Language: En Journal: J Neuromuscul Dis Year: 2024 Document type: Article Country of publication: Países Bajos