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Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy.
Kichula, Elizabeth A; Proud, Crystal M; Farrar, Michelle A; Kwon, Jennifer M; Saito, Kayoko; Desguerre, Isabelle; McMillan, Hugh J.
Affiliation
  • Kichula EA; Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
  • Proud CM; Children's Hospital of the King's Daughters, Norfolk, Virginia, USA.
  • Farrar MA; School of Women's and Children's Health, UNSW Medicine, University of New South Wales Sydney and Sydney Children's Hospital Network, Sydney, New South Wales, Australia.
  • Kwon JM; School of Medicine and Public Health, University of Wisconsin, Madison, Wisconsin, USA.
  • Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
  • Desguerre I; Necker-Enfants Malades Hospital, University of Paris, AP-HP, Paris, France.
  • McMillan HJ; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, Ontario, Canada.
Muscle Nerve ; 64(4): 413-427, 2021 Oct.
Article in En | MEDLINE | ID: mdl-34196026
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Full text: 1 Collection: 01-internacional Health context: 11_ODS3_cobertura_universal Database: MEDLINE Main subject: Biological Products / Recombinant Fusion Proteins / Muscular Atrophy, Spinal / Genetic Therapy / Expert Testimony / Clinical Decision-Making Type of study: Guideline / Prognostic_studies Limits: Humans Language: En Journal: Muscle Nerve Year: 2021 Document type: Article
Fulltext
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PubMed Links
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Full text: 1 Collection: 01-internacional Health context: 11_ODS3_cobertura_universal Database: MEDLINE Main subject: Biological Products / Recombinant Fusion Proteins / Muscular Atrophy, Spinal / Genetic Therapy / Expert Testimony / Clinical Decision-Making Type of study: Guideline / Prognostic_studies Limits: Humans Language: En Journal: Muscle Nerve Year: 2021 Document type: Article