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Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis: A phase 2, open label, multicenter study.
Guffon, Nathalie; Konstantopoulou, Vassiliki; Hennermann, Julia B; Muschol, Nicole; Bruno, Irene; Tummolo, Albina; Ceravolo, Ferdinando; Zardi, Giulia; Ballabeni, Andrea; Lund, Allan.
Afiliación
  • Guffon N; Reference Centre of Inherited Metabolic Diseases, CERLYMM, HCL, Lyon, France.
  • Konstantopoulou V; Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria.
  • Hennermann JB; Center for Pediatric and Adolescent Medicine, Villa Metabolica, University Medical Center Mainz, Mainz, Germany.
  • Muschol N; Department of Pediatrics, International Center for Lysosomal Disorders (ICLD), University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Bruno I; Institute for Maternal and Child Health-IRCCS Burlo Garofolo, Trieste, Italy.
  • Tummolo A; Department of Metabolic Diseases and Clinical Genetics, Bari, Italy.
  • Ceravolo F; Chiesi Farmaceutici, Parma, Italy.
  • Zardi G; CROS NT S.r.l, Verona, Italy.
  • Ballabeni A; Chiesi Farmaceutici, Parma, Italy.
  • Lund A; Centre for Inherited Metabolic Diseases, Department of Paediatrics and Adolescent Medicine and Department of Clinical Genetics, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
J Inherit Metab Dis ; 46(4): 705-719, 2023 07.
Article en En | MEDLINE | ID: mdl-36849760
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Alfa-Manosidosis Tipo de estudio: Clinical_trials Aspecto: Patient_preference Límite: Adolescent / Adult / Child / Child, preschool / Humans / Male Idioma: En Revista: J Inherit Metab Dis Año: 2023 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Alfa-Manosidosis Tipo de estudio: Clinical_trials Aspecto: Patient_preference Límite: Adolescent / Adult / Child / Child, preschool / Humans / Male Idioma: En Revista: J Inherit Metab Dis Año: 2023 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Estados Unidos