Safety and efficacy findings in the first-in-human trial (FIH) of the oral splice modulator branaplam in type 1 spinal muscular atrophy (SMA): interim results

著录项

• 来源
  《Neuromuscular disorders: NMD》 |2017年第2期|共2页
• 作者

  Charnas L.; Voltz E.; Pfister C.; Peters T.; Hartmann A.; Berghs-Clairmont C.; Praestgaard J.; de Raspide M.; Deconinck N.; Born A.; Baranello G.; Bertini E.; Schara U.; Goemans N.; Roubenoff R.;

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• 作者单位

  Novartis Inst Biomed Res Cambridge MA USA;

  Novartis Inst Biomed Res Basel Switzerland;

  Novartis Inst Biomed Res Basel Switzerland;

  Novartis Inst Biomed Res Basel Switzerland;

  Novartis Inst Biomed Res Basel Switzerland;

  Novartis Inst Biomed Res Cambridge MA USA;

  Novartis Pharmaceut E Hanover NJ USA;

  Novartis Pharmaceut Basel Switzerland;

  Univ Ziekenhius Ghent Belgium;

  Rigshosp Copenhagen Denmark;

  IRCCS Ist Neurol Carlo Besta Milan Italy;

  IRCCS Osped Pediat Bambino Gesu Rome Italy;

  Univ Klinikum Essen Essen Germany;

  Univ Ziekenhuis Leuven Leuven Belgium;

  Novartis Inst Biomed Res Basel Switzerland;

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• 原文格式 PDF
• 正文语种 eng
• 中图分类 神经病学;
• 关键词