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Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.
D'Silva, Arlene M; Holland, Sandra; Kariyawasam, Didu; Herbert, Karen; Barclay, Peter; Cairns, Anita; MacLennan, Suzanna C; Ryan, Monique M; Sampaio, Hugo; Smith, Nicholas; Woodcock, Ian R; Yiu, Eppie M; Alexander, Ian E; Farrar, Michelle A.
Afiliación
  • D'Silva AM; Department of Neurology, Sydney Children's Hospital Network, Sydney, New South Wales, Australia.
  • Holland S; School of Clinical Medicine, UNSW Medicine & Health, Randwick Clinical Campus, Discipline of Paediatrics, University of New South Wales, Sydney, New South Wales, Australia.
  • Kariyawasam D; Department of Neurology, Sydney Children's Hospital Network, Sydney, New South Wales, Australia.
  • Herbert K; Department of Neurology, Sydney Children's Hospital Network, Sydney, New South Wales, Australia.
  • Barclay P; School of Clinical Medicine, UNSW Medicine & Health, Randwick Clinical Campus, Discipline of Paediatrics, University of New South Wales, Sydney, New South Wales, Australia.
  • Cairns A; Physiotherapy, Sydney Children's Hospital, Sydney, New South Wales, Australia.
  • MacLennan SC; Department of Pharmacy, Prince of Wales Hospital, Sydney Children's Hospital Randwick and The Royal Hospital for Women, Randwick, New South Wales, Australia.
  • Ryan MM; Neurosciences Department, Queensland's Children Hospital, South Brisbane, Queensland, Australia.
  • Sampaio H; Neurology Department, Women's & Children's Hospital, North Adelaide, South Australia, Australia.
  • Smith N; School of Paediatrics and Reproductive Health, University of Adelaide, Adelaide, South Australia, Australia.
  • Woodcock IR; Department of Neurology, Royal Children's Hospital, Melbourne, Australia.
  • Yiu EM; Neuroscience Research Group, Murdoch Children's Research Institute, Melbourne, Australia.
  • Alexander IE; Department of Paediatrics, The University of Melbourne, Melbourne, Australia.
  • Farrar MA; Department of Neurology, Sydney Children's Hospital Network, Sydney, New South Wales, Australia.
Ann Clin Transl Neurol ; 9(3): 339-350, 2022 03.
Article en En | MEDLINE | ID: mdl-35170254

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinales de la Infancia / Efectos Colaterales y Reacciones Adversas Relacionados con Medicamentos Tipo de estudio: Observational_studies / Prognostic_studies Límite: Child / Child, preschool / Humans / Infant / Newborn País/Región como asunto: Oceania Idioma: En Revista: Ann Clin Transl Neurol Año: 2022 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinales de la Infancia / Efectos Colaterales y Reacciones Adversas Relacionados con Medicamentos Tipo de estudio: Observational_studies / Prognostic_studies Límite: Child / Child, preschool / Humans / Infant / Newborn País/Región como asunto: Oceania Idioma: En Revista: Ann Clin Transl Neurol Año: 2022 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Estados Unidos