Anti-inflammatory properties in cellular models of neurodegenerative diseases.Decrease of nitrite release after LPS stimulation.Efficacy in preclinical models of multiple sclerosis and fragile X syndrome.Improvement of delayed myogenesis in CDM1 myoblasts and neuroprotection properties in SMA-derived cells [20,32,119,120,121]. The gene discussed is SMN1; the disease is fragile X syndrome.