Results of a Phase II Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3- to 25-Year-Old Spinal Muscular Atrophy Patients

Background and Objectives: Olesoxime (TRO19622) was identified as a potential treatment of spinal muscular atrophy (SMA) based on its beneficial effects in multiple preclinical neurodegeneration models. Olesoxime promotes neuron survival, neurite outgrowth, recovery from nerve injury and accelerates myelination or remyelination in models of demyelinating diseases. Maintaining motor neuron architecture and survival is highly relevant to SMA, a disease associated with progressive motor neuron compromise mainly affecting neuromuscular function. A clinical study has been performed to evaluate the effects of olesoxime treatment in patients with SMA.