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The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
Harmatz, P; Hendriksz, C J; Lampe, C; McGill, J J; Parini, R; Leão-Teles, E; Valayannopoulos, V; Cole, T J; Matousek, R; Graham, S; Guffon, N; Quartel, A.
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  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA. Electronic address: pharmatz@mail.cho.org.
  • Hendriksz CJ; University of Pretoria, The Mark Holland Metabolic Unit, Salford Royal Foundation NHS Trust, Salford, UK.
  • Lampe C; Department of Pediatric and Adolescent Medicine, Villa Metabolica, University Medical Center of the University of Mainz, Germany.
  • McGill JJ; Department of Metabolic Medicine, Lady Cilento Children's Hospital, South Brisbane, QLD, Australia.
  • Parini R; UOS Malattie Metaboliche Rare, Clinica Pediatrica Ospedale San Gerardo, Monza, Italy.
  • Leão-Teles E; Unidade Doenças Metabólicas, Hospital Pediátrico Integrado, Centro Hospitalar de S. João, Porto, Portugal.
  • Valayannopoulos V; Reference Center for Inherited Metabolic Diseases and IMAGINE Institute, Necker-Enfants Malades Hospital, Paris, France.
  • Cole TJ; Population, Policy and Practice Programme, UCL Institute of Child Health, London, UK.
  • Matousek R; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Graham S; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Guffon N; Reference Center for Inherited Metabolic Diseases, Hôpital Femme Mère Enfant, Lyon, France.
  • Quartel A; BioMarin Pharmaceutical Inc., Novato, CA, USA.
Mol Genet Metab ; 122(1-2): 107-112, 2017 09.
Article en En | MEDLINE | ID: mdl-28457718
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Estatura / Mucopolisacaridosis VI / N-Acetilgalactosamina-4-Sulfatasa / Terapia de Reemplazo Enzimático Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2017 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Estatura / Mucopolisacaridosis VI / N-Acetilgalactosamina-4-Sulfatasa / Terapia de Reemplazo Enzimático Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2017 Tipo del documento: Article Pais de publicación: Estados Unidos