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Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy.
Garcia, Paula; Phillips, Dawn; Johnson, JoAnn; Martin, Kenneth; Randolph, Linda M; Rosenfeld, Howard; Harmatz, Paul.
Afiliação
  • Garcia P; Hospital Pediátrico de Coimbra, Coimbra, Portugal.
  • Phillips D; UNC Chapel Hill Division of Physical Therapy, School of Medicine, Chapel Hill, NC, USA.
  • Johnson J; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Martin K; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Randolph LM; Children's Hospital Los Angeles, CA, USA.
  • Rosenfeld H; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA. Electronic address: Paul.harmatz@ucsf.edu.
Mol Genet Metab ; 133(1): 100-108, 2021 05.
Article em En | MEDLINE | ID: mdl-33775523
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Condroitina Sulfatases / Mucopolissacaridose VI / N-Acetilgalactosamina-4-Sulfatase / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child / Child, preschool / Humans / Infant / Male Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Condroitina Sulfatases / Mucopolissacaridose VI / N-Acetilgalactosamina-4-Sulfatase / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child / Child, preschool / Humans / Infant / Male Idioma: En Ano de publicação: 2021 Tipo de documento: Article