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Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial.
Borgwardt, Line; Guffon, Nathalie; Amraoui, Yasmina; Dali, Christine I; De Meirleir, Linda; Gil-Campos, Mercedes; Heron, Bénédicte; Geraci, Silvia; Ardigò, Diego; Cattaneo, Federica; Fogh, Jens; Van den Hout, J M Hannerieke; Beck, Michael; Jones, Simon A; Tylki-Szymanska, Anna; Haugsted, Ulla; Lund, Allan M.
Afiliação
  • Borgwardt L; Department of Paediatrics and Adolescent Medicine, Centre for Inherited Metabolic Diseases, Copenhagen, Denmark. Line.Gutte.Borgwardt@regionh.dk.
  • Guffon N; Center for Genomic Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark. Line.Gutte.Borgwardt@regionh.dk.
  • Amraoui Y; Department of Clinical Genetics, Centre for Inherited Metabolic Diseases, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark. Line.Gutte.Borgwardt@regionh.dk.
  • Dali CI; Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Femme Mère Enfant, Lyon, France.
  • De Meirleir L; Center for Pediatric and Adolescent Medicine, University Medical Center Mainz, Villa Metabolica, Mainz, Germany.
  • Gil-Campos M; Department of Paediatrics and Adolescent Medicine, Centre for Inherited Metabolic Diseases, Copenhagen, Denmark.
  • Heron B; Paediatric Neurology and Metabolism, Universitair Ziekenhuis, Brussel, Belgium.
  • Geraci S; Unidad de Metabolismo e Investigación Pediátrica, Hospital Universitario Reina Sofía, IMIBIC, Universidad de Córdoba, CIBERObn, Córdoba, Spain.
  • Ardigò D; Department of Pediatric Neurology, Reference Center for Lysosomal Diseases, Trousseau Hospital, APHP, and GRC ConCer-LD, Sorbonne Universities, UPMC University 06, Paris, France.
  • Cattaneo F; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Fogh J; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Van den Hout JMH; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Beck M; Zymenex A/S, Hillerød, Denmark.
  • Jones SA; Center for Lysosomal and Metabolic Diseases (Department of Pediatrics), Erasmus MC University Medical Center - Sophia Children's Hospital, Rotterdam, The Netherlands.
  • Tylki-Szymanska A; Institute of Human Genetics, University Medical Center, Mainz, Germany.
  • Haugsted U; Manchester Centre for Genomic Medicine, Central Manchester University Hospitals NHS Foundation Trust, Manchester, UK.
  • Lund AM; Department of Paediatric, Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland.
J Inherit Metab Dis ; 41(6): 1215-1223, 2018 11.
Article em En | MEDLINE | ID: mdl-29846843
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Alfa-Manosidase / Terapia de Reposição de Enzimas / Alfa-Manosidose Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male País/Região como assunto: Europa Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2018 Tipo de documento: Article País de afiliação: Dinamarca
Texto completo
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XML
PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Alfa-Manosidase / Terapia de Reposição de Enzimas / Alfa-Manosidose Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male País/Região como assunto: Europa Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2018 Tipo de documento: Article País de afiliação: Dinamarca