G.P.2.16 Valproate, acetylcarnitine, folic acid and vitamin B12 in spinal muscular atrophy: Preliminary data of an open-label study in children

In a preliminary open-label study from 2007 involving 15 children with Spinal Muscular Atrophy (SMA) types II and III, researchers investigated the tolerability and efficacy of a treatment regimen consisting of valproate, acetylcarnitine, folic acid, and vitamin B12 over a one-year period. The participants, aged between 3 years and 10 months to 12 years and 1 month, were assessed for changes in motor function, muscle strength, and forced vital capacity at 6 and 12 months. After six months, the patients showed increased muscle strength and improved subjective function without significant adverse effects, including weight, hair loss, gastric, and metabolic tolerance. The study suggested that long-term treatment with this combination may be beneficial for SMA patients, with no significant adverse effects noted at the time of reporting.