Original article| Volume 11, ISSUE 6, P337-340, November 2007

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


      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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        • Gorospe J.R.
        • Tharp M.D.
        • Hinckley J.
        • Kornegay J.N.
        • Hoffman E.P.
        A role for mast cells in the progression of Duchenne muscular dystrophy? Correlations in dystrophin-deficient humans, dogs, and mice.
        J Neurol Sci. 1994; 122: 44-56
        • Gorospe J.R.
        • Nishikawa B.K.
        • Hoffman E.P.
        Recruitment of mast cells to muscle after mild damage.
        J Neurol Sci. 1996; 135: 10-17
        • Granchelli J.A.
        • Pollina C.
        • Hudecki M.S.
        Duchenne-like myopathy in double-mutant mdx mice expressing exaggerated mast cell activity.
        J Neurol Sci. 1995; 131: 1-7
        • Gorospe R.J.
        • Tharp M.
        • Demitsu T.
        • Hoffman E.P.
        Dystrophin-deficient myofibers are vulnerable to mast cell granule-induced necrosis.
        Neuromuscul Disord. 1994; 4: 325-333
        • Yoshizawa I.
        • Nakajima T.
        • Kawano Y.
        • Noma T.
        Suppressive effect of oxatomide on the induction of IL2 responsiveness by DF-stimulated lymphocytes from patients with bronchial asthma.
        Arerugi. 1994; 43: 535-543
        • Chen Y.W.
        • Zhao P.
        • Borup R.
        • Hoffman E.P.
        Expression profiling in the muscular dystrophies: identification of novel aspects of molecular pathophysiology.
        J Cell Biol. 2000; 151: 1321-1336
        • Granchelli J.A.
        • Pollina C.
        • Hudecki M.S.
        Pre-clinical screening of drugs using the mdx mouse.
        Neuromuscul Disord. 2000; 10: 235-239
        • Escolar D.
        • Henricson E.
        • Mayhew J.
        • et al.
        Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children.
        Muscle Nerve. 2001; 24: 787-793
        • Mayhew J.E.
        • Florence J.M.
        • Mayhew T.P.
        • et al.
        Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy.
        Muscle Nerve. 2007; 35: 36-42
        • Escolar D.M.
        • Buyse G.M.
        • Henricson E.
        • et al.
        CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.
        Ann Neurol. 2005; 58: 151-155
        • Verbeke G.
        • Molenberghs G.
        Linear mixed models for longitudinal data.
        Springer, New York2000
        • Chen Y.-W.
        • Nagaraju K.
        • Bakay M.
        • et al.
        Early onset of inflammation and later involvement of TGFβ in Duchenne muscular dystrophy.
        Neurology. 2005; 65: 826-834