Spinal Muscular Atrophy Clinical Trials: Lessons Learned

S. T. Iannaccone, L. L. Nelson

Research output: Chapter in Book/Report/Conference proceedingChapter

2 Scopus citations


There are few completed randomized clinical trials for spinal muscular atrophy (SMA). There is no known effective treatment and most trials have been open label. However, much has been learned from those negative trials that helped improve trial design so that future studies will be powered and valid. Lessons learned include the fact that easy fatigability influences a subject's performance on outcome measures and that this fatigue can be induced by prolonged travel to the study site and prolonged procedures during the study visit. There is new and concerning use of social media by subjects and their families that could affect enrollment and outcomes; this demands new guidelines for trial participants and for investigators. Finally, it is very clear that all study groups must have received standard of care at the time of enrollment and throughout the study in order to avoid confounders of outcome measures.

Original languageEnglish (US)
Title of host publicationSpinal Muscular Atrophy
Subtitle of host publicationDisease Mechanisms and Therapy
PublisherElsevier Inc.
Number of pages6
ISBN (Electronic)9780128036860
ISBN (Print)9780128036853
StatePublished - Jan 1 2017


  • Cochrane reviews
  • Fatigability
  • Outcome measures
  • Social media
  • Standard of care

ASJC Scopus subject areas

  • General Medicine
  • General Neuroscience


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