Global treatment responder analysis demonstrates clinically relevant effect of velmanase alfa long term enzyme replacement therapy for alpha mannosidosis, in a phase III randomized placebo controlled trial

著录项

• 来源
  《Molecular genetics and metabolism》 |2018年第2期|共1页
• 作者

  Harmatz Paul; Cattaneo Federica; Ardigo Diego; Geraci Silvia; Amraoui Yasmina; Gil-Campos Mercedes; De Meirleir Linda; Muschol Nicole; Tylki-Szymanska Anna; Wijburg Frits; Van den Hout Johanna M. P.; Guffon Nathalie; Dali Christine I.; Jones Simon A.; Lund Allan M.; Borgwardt Line;

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• 作者单位

  Childrens Hosp Oakland Oakland CA USA;

  Chiesi Framaceut SpA Parma Italy;

  Chiesi Framaceut SpA Parma Italy;

  Chiesi Framaceut SpA Parma Italy;

  Univ Med Ctr Mainz Ctr Pediat &

  Adolescent Med Villa Metab Mainz Germany;

  Univ Cordoba Unidad Metab &

  Invest Pediat Hosp Univ Reina Sofia IMIBIC CIBERObn Cordoba Spain;

  Univ Ziekenhuis Paediat Neurol &

  Metab Brussels Belgium;

  Univ Med Ctr Hamburg Eppendorf Int Ctr Lysosomal Disorders Hamburg Germany;

  Childrens Mem Hlth Inst Dept Paediat Nutr &

  Metab Dis Warsaw Poland;

  Acad Med Ctr Amsterdam Netherlands;

  Erasmus MC Univ Med Ctr Sophia Childrens Hosp Ctr Lysosomal &

  Metab Dis Dept Pediat Rotterdam;

  Hop Femme Mere Enfant Ctr Reference Malad Hereditaires Metab Lyon France;

  Rigshosp Copenhagen Univ Hosp Ctr Inherited Metab Dis Dept Clin Genet Copenhagen Denmark;

  Cent Manchester Univ Hosp NHS Fdn Trust Manchester Ctr Genom Med Manchester Lancs England;

  Rigshosp Copenhagen Univ Hosp Ctr Inherited Metab Dis Dept Clin Genet Copenhagen Denmark;

  Rigshosp Copenhagen Univ Hosp Ctr Inherited Metab Dis Dept Clin Genet Copenhagen Denmark;

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• 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