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Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
Lund, Allan M; Borgwardt, Line; Cattaneo, Federica; Ardigò, Diego; Geraci, Silvia; Gil-Campos, Mercedes; De Meirleir, Linda; Laroche, Cécile; Dolhem, Philippe; Cole, Duncan; Tylki-Szymanska, Anna; Lopez-Rodriguez, Monica; Guillén-Navarro, Encarna; Dali, Christine I; Héron, Bénédicte; Fogh, Jens; Muschol, Nicole; Phillips, Dawn; Van den Hout, J M Hannerieke; Jones, Simon A; Amraoui, Yasmina; Harmatz, Paul; Guffon, Nathalie.
  • Lund AM; Departments of Paediatrics and Adolescent Medicine, Centre for Inherited Metabolic Diseases, Copenhagen, Denmark.
  • Borgwardt L; Department of Clinical Genetics, Centre for Inherited Metabolic Diseases, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Cattaneo F; Departments of Paediatrics and Adolescent Medicine, Centre for Inherited Metabolic Diseases, Copenhagen, Denmark. Line.Gutte.Borgwardt@regionh.dk.
  • Ardigò D; Department of Clinical Genetics, Centre for Inherited Metabolic Diseases, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark. Line.Gutte.Borgwardt@regionh.dk.
  • Geraci S; Center for Genomic Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark. Line.Gutte.Borgwardt@regionh.dk.
  • Gil-Campos M; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • De Meirleir L; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Laroche C; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Dolhem P; Unidad de Metabolismo e Investigación Pediátrica, Hospital Universitario Reina Sofía, IMIBIC, Universidad de Córdoba, CIBERObn, Córdoba, Spain.
  • Cole D; Paediatric Neurology and Metabolism, Universitair Ziekenhuis, Brussels, Belgium.
  • Tylki-Szymanska A; Limoges Hospital, Limoges, France.
  • Lopez-Rodriguez M; Centre Hospitalier de Saint-Quentin, Saint-Quentin, France.
  • Guillén-Navarro E; Department of Medical Biochemistry and Immunology, University Hospital of Wales, Cardiff, Wales, UK.
  • Dali CI; Department of Paediatric, Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland.
  • Héron B; Hospital Central Cruz Roja, Madrid, Spain.
  • Fogh J; Medical Genetics Section, Hospital Clínico Universitario Virgen de la Arrixaca, IMIB-Arrixaca, CIBERER-ISCIII, Madrid, Spain.
  • Muschol N; Departments of Paediatrics and Adolescent Medicine, Centre for Inherited Metabolic Diseases, Copenhagen, Denmark.
  • Phillips D; Service de Neuropédiatrie, Centre de Référence des Maladies Lysosomales, and Sorbonne Université, GRC n°19, pathologies Congénitales du Cervelet-LeucoDystrophies, AP-HP, Hôpital Armand Trousseau, F-75012, Paris, France.
  • Van den Hout JMH; Zymenex A/S, Hillerød, Denmark.
  • Jones SA; International Center for Lysosomal Disorders, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Amraoui Y; Evidera, Bethesda, MD, USA.
  • Harmatz P; Center for Lysosomal and Metabolic Diseases (department of Pediatrics), Erasmus MC University Medical Center - Sophia Children's Hospital, Rotterdam, The Netherlands.
  • Guffon N; Manchester Centre for Genomic Medicine, Central Manchester University Hospitals NHS Foundation Trust, Manchester, UK.
J Inherit Metab Dis ; 41(6): 1225-1233, 2018 11.
Article en En | MEDLINE | ID: mdl-29725868
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Alfa-Manosidasa / Terapia de Reemplazo Enzimático / Alfa-Manosidosis Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Child / Female / Humans / Male País como asunto: Europa Idioma: En Año: 2018 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Alfa-Manosidasa / Terapia de Reemplazo Enzimático / Alfa-Manosidosis Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Child / Female / Humans / Male País como asunto: Europa Idioma: En Año: 2018 Tipo del documento: Article