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Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials.
Schreiber A, Brochard S, Rippert P, Fontaine-Carbonnel S, Payan C, Poirot I, Hamroun D, Vuillerot C; MFM DMD Corticosteroids Group. Schreiber A, et al. Among authors: fontaine carbonnel s. Dev Med Child Neurol. 2018 Feb;60(2):185-191. doi: 10.1111/dmcn.13590. Epub 2017 Oct 9. Dev Med Child Neurol. 2018. PMID: 28990163 Free article.
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial.
Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators. Bertini E, et al. Lancet Neurol. 2017 Jul;16(7):513-522. doi: 10.1016/S1474-4422(17)30085-6. Epub 2017 Apr 28. Lancet Neurol. 2017. PMID: 28460889 Free article. Clinical Trial.