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 
Lancet Neurol. 2017. 16(7):513-522.
This phase 2 randomised, double-blind, placebo-controlled study was done in 22 neuromuscular care centres from 7 european country aimed to study safety and efficacy of olesoxime in patients aged 3-25 years with type 2 or type 3 SMA.
The primary outcome measure was change from baseline compared with 24 months of MFM D1+D2 scores.
Olesoxime seemed to be safe and generally well tolerated at the doses studied. Results suggested that olesoxime might maintain motor function in patients with type 2 or type 3 SMA over a period of 24 months

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Keywords: Spinal Muscular atrophy, Phase 2, Safety, Olesoxime

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