Efficacy and safety of intrathecal idursulfase in pediatric patients with mucopolysaccharidosis type II and early cognitive impairment: Design and methods of a controlled, randomized, phase II/III multicenter study

著录项

• 来源
  《Molecular genetics and metabolism》 |2018年第2期|共2页
• 作者

  Muenzer Joseph; Burton Barbara K.; Harmatz Paul; Gonzalez Gutierre-Solana Luis; Ruiz-Garcia Matilde; Jones Simon A.; Guffon Nathalie; Inbar-Feigenberg Michal; Bratkovic Drago; Wu Yuna; Alexanderian David;

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• 作者单位

  Univ North Carolina Chapel Hill Chapel Hill NC USA;

  Northwestern Univ Ann &

  Robert H Lurie Childrens Hosp Chicago Chicago IL 60611 USA;

  UCSF Benioff Childrens Hosp Oakland Oakland CA USA;

  Hosp Infantil Nino Jesus Madrid Spain;

  Natl Inst Pediat Mexico City DF Mexico;

  Cent Manchester Univ Hosp NHS Fdn Trust Manchester &

  Acad Hlth Sci Ctr St Marys Hosp Manchester;

  Hop Femme Mere Enfant Bron France;

  Univ Toronto Hosp Sick Children Toronto ON Canada;

  Womens &

  Childrens Hosp Adelaide SA Australia;

  Shire Lexington MA USA;

  Shire Lexington MA USA;

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• 收录信息
• 原文格式 PDF
• 正文语种 eng
• 中图分类 内分泌腺疾病及代谢病;
• 关键词