- CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy.
CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy.
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society (2007-04-27)
Gunnar M Buyse, Nathalie Goemans, Erik Henricson, Alejandro Jara, Marleen van den Hauwe, Robert Leshner, Julaine M Florence, Jill E Mayhew, Diana M Escolar
PMID17459739
摘要
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.
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