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Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial.
Hendriksz, Christian J; Giugliani, Roberto; Harmatz, Paul; Mengel, Eugen; Guffon, Nathalie; Valayannopoulos, Vassili; Parini, Rossella; Hughes, Derralynn; Pastores, Gregory M; Lau, Heather A; Al-Sayed, Moeenaldeen D; Raiman, Julian; Yang, Ke; Mealiffe, Matthew; Haller, Christine.
Afiliação
  • Hendriksz CJ; Salford Royal NHS Foundation Trust, Salford, UK. Electronic address: chris.hendriksz@srft.nhs.uk.
  • Giugliani R; Medical Genetics Service/HCPA, Department of Genetics/UFRGS and INAGEMP, Porto Alegre, Brazil.
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Mengel E; Villa Metabolica, Centre for Pediatric and Adolescent Medicine, MC University of Mainz, Mainz, Germany.
  • Guffon N; Hôpital Femme Mère Enfant Centre de référence des maladies héréditaires du metabolism, Bron Cedex, France.
  • Valayannopoulos V; Reference Center for Inherited Metabolic Disease, Hôpital Necker-Enfants Malades and IMAGINE Institute, Paris, France.
  • Parini R; Unita Operativa Semplice Malattie Metaboliche Rare, Azienda Ospedaliera San Gerardo, Monza, Italy.
  • Hughes D; Royal Free London NHS Foundation Trust & University College, London, UK.
  • Pastores GM; New York University School of Medicine, New York, NY, USA.
  • Lau HA; New York University School of Medicine, New York, NY, USA.
  • Al-Sayed MD; King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.
  • Raiman J; Hospital for Sick Children, Toronto, ON, Canada.
  • Yang K; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Mealiffe M; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Haller C; BioMarin Pharmaceutical Inc., Novato, CA, USA.
Mol Genet Metab ; 114(2): 178-85, 2015 Feb.
Article em En | MEDLINE | ID: mdl-25284089
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Condroitina Sulfatases / Mucopolissacaridose IV / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Child / Child, preschool / Humans / Middle aged Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2015 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 IV / Terapia de Reposição de Enzimas Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Child / Child, preschool / Humans / Middle aged Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2015 Tipo de documento: Article