AVXS-101 phase 1 gene replacement therapy clinical trial in spinal muscular atrophy type 1 (SMA1): 24-month event-free survival and achievement of developmental milestones

著录项

• 来源
  《Human gene therapy》 |2018年第12期|共1页
• 作者

  Mendell J. R.; Al-Zaidy S.; Shell R.; Arnold W. D.; Rodino-Klapac L.; Prior T. W.; Lowes L. P.; Alfano L. N.; Berry K.; Church K.; Kissel J. T.; Nagendran S.; LItalien J.; Sproule D. M.; Wells C.; Burghes A. H. M.; Foust K. D.; Meyer K.; Likhite S.; Kaspar B. K.;

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• 作者单位

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Dept Pediat Sect Pulm Med Columbus OH USA;

  Ohio State Univ Dept Neurol Columbus OH 43210 USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Ohio State Univ Dept Pathol Columbus OH 43210 USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Ohio State Univ Dept Pediat Columbus OH 43210 USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  Ohio State Univ Dept Neurol Columbus OH 43210 USA;

  AveXis Inc Bannockburn IL USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

  Nationwide Childrens Hosp Ctr Gene Therapy Columbus OH USA;

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• 收录信息
• 原文格式 PDF
• 正文语种 eng
• 中图分类 治疗学;
• 关键词