Buyse Gunnar M, Goemans Nathalie, Henricson Erik, Jara Alejandro, van den Hauwe Marleen, Leshner Robert, Florence Julaine M, Mayhew Jill E, Escolar Diana M
University Hospitals KU Leuven, Leuven, Herestraat 49, B-3000 Leuven, Belgium.
Eur J Paediatr Neurol. 2007 Nov;11(6):337-40. doi: 10.1016/j.ejpn.2007.02.009. Epub 2007 Apr 24.
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.
作者报告了一项针对14名年龄在5至10岁、未使用过类固醇的杜氏肌营养不良症（DMD）男孩的奥沙米特开放标签两中心治疗试验。在3个月的无药物导入期和6个月的治疗期之间进行线性进展比较，结果显示在肌肉力量的定量（QMT）和手动（MMT）测量以及定时功能测试方面没有显著差异。但不能排除随着时间推移力量衰退有适度缓解的情况。
