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Follow-Up Study of Olesoxime in Patients with Type 2 or Non-Ambulatory Type 3 Spinal Muscular Atrophy

New results of the Long Term Study of Oleos (NCT02628743)

olesoxime was generally safe and well tolerated at the dose assessed. Maintenance of motor function observed over 2 years in the Phase 2 study was followed by a substantial decline in MFM D1+D2 (>2 points/year) after drug discontinuation. However, the ~2-point MFM treatment difference between olesoxime and placebo at the end of Phase 2 was maintained at OLEOS baseline. Furthermore, olesoxime open-label treatment stabilized motor function (mean change in MFM D1 + D2 from baseline: 6 months, −0.03 [SD, 4.79; n=124]; 12 months, −0.22 [SD, 4.74, n=104]). These data support the long-term stabilization of motor function observed in the Phase 2 study

Published inOlesoxime (TRO19622)Spinal Muskelatrofi