Growing evidence suggests mitochondrial dysfunction is a key disease mechanism and valid therapeutic target in spinal muscular atrophy (SMA). Olesoxime is an orally-administered, mitochondria-targeting compound that promotes survival and function of motor neurons, muscle and other cell types. This study assessed the efficacy, safety and tolerability of olesoxime in patients aged 3–25 years with Type 2 or non-ambulatory Type 3 SMA (NCT01302600). Participants were eligible for a follow-up, open-label study (OLEOS; NCT02628743) to assess the long-term safety and efficacy of olesoxime.

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