Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG)

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• 来源
  《Neuromuscular disorders: NMD》 |2019年第1期|共1页
• 作者

  Finkel R.; Day J.; Darras B.; Kuntz N.; Connolly A.; Crawford T.; Butterfield R.; Shieh P.; Tennekoon G.; Iannaccone S.; Ogrinc F.; Kavanagh S.; Kernbauer E.; Whittle J.; LItalien J.; Kaspar B.; Sproule D.; Spector S.; Feltner D.; Mendell J.;

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• 作者单位

  Nemours Childrens Hosp Orlando FL USA;

  Stanford Univ Stanford CA 94305 USA;

  Boston Childrens Hosp Boston MA USA;

  Lurie Childrens Hosp Chicago IL USA;

  WashU St Louis St Louis MO USA;

  Johns Hopkins Univ Baltimore MD USA;

  Univ Utah Hlth Salt Lake City UT USA;

  Univ Calif Los Angeles Sch Med Los Angeles CA USA;

  CHOP Philadelphia PA USA;

  UT Southwestern Med Ctr Dallas TX USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  AveXis Inc Bannockburn IL USA;

  Nationwide Childrens Hosp 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 神经病学;
• 关键词