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Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study.
Landfeldt, Erik; Zhang, Rongrong; Childs, Anne-Marie; Johannsen, Jessika; O'Rourke, Declan; Sejersen, Thomas; Strautmanis, Jurgis; Schara-Schmidt, Ulrike; Tulinius, Mar; Walter, Maggie C; Willis, Tracey; Buesch, Katharina.
Afiliação
  • Landfeldt E; IQVIA, Stockholm, Sweden.
  • Zhang R; PTC Therapeutics Sweden AB, Askim, Sweden.
  • Childs AM; Leeds Teaching Hospitals, Leeds, UK.
  • Johannsen J; Department of Pediatrics, Neuropediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • O'Rourke D; Department of Neurology, Children's Health Ireland at Temple Street, Dublin, Ireland.
  • Sejersen T; Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden.
  • Strautmanis J; Department of Neuropediatrics, Astrid Lindgren Children´s Hospital, Karolinska University Hospital, Stockholm, Sweden.
  • Schara-Schmidt U; Department of Neurology and Neurosurgery, Children's Clinical University Hospital, Riga, Latvia.
  • Tulinius M; Department of Neuropediatrics, Developmental Neurology and Social Pediatrics, Centre for Neuromuscular Disorders in Children and Adolescents, Children´s University Clinic Essen, University of Duisburg-Essen, Essen, Germany.
  • Walter MC; Department of Pediatrics, University of Gothenburg, Gothenburg, Sweden.
  • Willis T; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.
  • Buesch K; Robert Jones and Agnes Hunt Hospital, Oswestry, UK.
J Med Econ ; 25(1): 808-816, 2022.
Article em En | MEDLINE | ID: mdl-35642753

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Prognostic_studies Aspecto: Patient_preference Limite: Child, preschool / Humans Idioma: En Revista: J Med Econ Assunto da revista: SERVICOS DE SAUDE Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Suécia

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Prognostic_studies Aspecto: Patient_preference Limite: Child, preschool / Humans Idioma: En Revista: J Med Econ Assunto da revista: SERVICOS DE SAUDE Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Suécia
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