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Published on: May 7, 2020
CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy
Gunnar M Buyse1, Nathalie Goemans, Erik Henricson
1University Hospitals KU Leuven, Leuven, Herestraat 49, B-3000 Leuven, Belgium. gunnar.buyse@uz.kuleuven.ac.be
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.

