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Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program.
Lampe, Christina; Harmatz, Paul R; Parini, Rossella; Sharma, Reena; Teles, Elisa Leão; Johnson, Julie; Sivam, Debbie; Sisic, Zlatko.
Afiliação
  • Lampe C; Department of Child Neurology, Justus-Liebig University, Gießen, Germany. Electronic address: Christina.Lampe72@gmail.com.
  • Harmatz PR; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA. Electronic address: pharmatz@mail.cho.org.
  • Parini R; Fondazione MBBM San Gerardo Hospital, Monza, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. Electronic address: rossella.parini@unimib.it.
  • Sharma R; Salford Royal Hospital NHS Foundation Trust, Salford, UK. Electronic address: Reena.Sharma@srft.nhs.uk.
  • Teles EL; São João Hospital, Porto, Portugal. Electronic address: e.leaoteles@gmail.com.
  • Johnson J; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: JJohnson@bmrn.com.
  • Sivam D; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: debbie.sivam@bmrn.com.
  • Sisic Z; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: Zlatko.Sisic@bmrn.com.
Mol Genet Metab ; 127(4): 355-360, 2019 08.
Article em En | MEDLINE | ID: mdl-31324526
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Sistema de Registros / Mucopolissacaridose VI / N-Acetilgalactosamina-4-Sulfatase / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Diagnostic_studies / Observational_studies / Screening_studies Limite: Adolescent / Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2019 Tipo de documento: Article
Texto completo
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Sistema de Registros / Mucopolissacaridose VI / N-Acetilgalactosamina-4-Sulfatase / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials / Diagnostic_studies / Observational_studies / Screening_studies Limite: Adolescent / Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2019 Tipo de documento: Article