Perspectives on clinical trials in spinal muscular atrophy

Kathryn J. Swoboda, John T. Kissel, Thomas O. Crawford, Mark B. Bromberg, Gyula Acsadi, Guy D'Anjou, Kristin J. Krosschell, Sandra P. Reyna, Mary K. Schroth, Charles B. Scott, Louise R. Simard

Research output: Contribution to journalReview articlepeer-review

48 Scopus citations


Spinal muscular atrophy is one of the most heterogeneous of the single-gene neuromuscular disorders. The broad spectrum of severity, with onset from the prenatal period to adulthood, presents unique challenges in the design and implementation of clinical trials. The clinical classification of subjects into severe (type 1), intermediate (type 2), and mild (type 3) subtypes has proved useful both in enhancing communication among clinicians internationally and in forging the collaborative development of outcome measures for clinical trials. Ideally, clinical trial design in spinal muscular atrophy must take into account the spinal muscular atrophy type, patient age, severity-of-affection status, nature of the therapeutic approach, timing of the proposed intervention relative to disease progression, and relative homogeneity of the cohort to be studied. Following is an overview of the challenges and opportunities, current and future therapeutic strategies, and progress to date in clinical trials in spinal muscular atrophy.

Original languageEnglish (US)
Pages (from-to)957-966
Number of pages10
JournalJournal of child neurology
Issue number8
StatePublished - Aug 2007


  • Clinical trials
  • Histone deacetylase inhibitors
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Clinical Neurology


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