CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

Gunnar M. Buyse, Nathalie Goemans, Erik Henricson, Alejandro Jara, Marleen van den Hauwe, Robert Leshner, Julaine M. Florence, Jill E. Mayhew, Diana M. Escolar

Research output: Contribution to journalArticlepeer-review

Abstract

The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5-10 years. Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.

Original languageEnglish (US)
Pages (from-to)337-340
Number of pages4
JournalEuropean Journal of Paediatric Neurology
Volume11
Issue number6
DOIs
StatePublished - Nov 2007

Keywords

  • Clinical trial
  • Duchenne
  • Muscle
  • Muscular dystrophy
  • Oxatomide
  • Treatment

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Clinical Neurology

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