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Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.
Lau, Heather A; Viskochil, David; Tanpaiboon, Pranoot; Lopez, Antonio Gonzalez-Meneses; Martins, Esmeralda; Taylor, Julie; Malkus, Betsy; Zhang, Lin; Jurecka, Agnieszka; Marsden, Deborah.
Afiliação
  • Lau HA; NYU Grossman School of Medicine, Department of Neurology, New York, NY, USA. Electronic address: Heather.Lau@nyulangone.org.
  • Viskochil D; University of Utah, Department of Pediatrics, Salt Lake City, UT, USA. Electronic address: Dave.Viskochil@hsc.utah.edu.
  • Tanpaiboon P; Rare Disease Institute, Children's National Health System, Washington, DC, USA. Electronic address: PTanpaib@childrensnational.org.
  • Lopez AG; Hospital Universitario Virgen del Rocío and Universidad de Sevilla, Seville, Spain. Electronic address: antonio.gonzalezmeneses.l.sspa@juntadeandalucia.es.
  • Martins E; Centro Hospitalar Universitário do Porto, Hospital de Santo António, Porto, Portugal. Electronic address: esmeralda.dia@chporto.min-saude.pt.
  • Taylor J; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: JTaylor@ultragenyx.com.
  • Malkus B; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: BMalkus@ultragenyx.com.
  • Zhang L; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: LZhang@ultragenyx.com.
  • Jurecka A; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: ajurecka@gmail.com.
  • Marsden D; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: DMarsden@ultragenyx.com.
Mol Genet Metab ; 136(1): 28-37, 2022 05.
Article em En | MEDLINE | ID: mdl-35331634
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose VII Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Child / Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2022 Tipo de documento: Article
Texto completo
Adicionar na Minha BVS
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose VII Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Child / Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2022 Tipo de documento: Article