Valproate, Acetylcarnitine, Folic Acid, and Vitamin B12 in Spinal Muscular Atrophy: Preliminary Data of an Open-Label Study in Children

    September 2007 in “ Neuromuscular disorders ”
    Alessandro Ghezzo, Francesco Danilo Tiziano, Raffaele Lodi, Caterina Tonon, Christina Brahe, Alessandro Pini
    TLDR The treatment with valproate, acetylcarnitine, folic acid, and vitamin B12 may improve muscle strength in children with SMA without significant side effects.
    This open-label study aimed to evaluate the tolerability and efficacy of a combination treatment involving valproate, acetylcarnitine, folic acid, and vitamin B12 in children with Spinal Muscular Atrophy (SMA) types II and III. Fifteen patients aged between 3 years and 10 months to 12 years and 1 month were enrolled. The treatment regimen included oral valproate (20 mg/kg per day), acetylcarnitine (1 g/day), with some patients also receiving folic acid (10 mg/week) and vitamin B12 (0.8 mg/week). Efficacy was measured through changes in motor function, muscle strength, and forced vital capacity over 6 and 12 months. Tolerability was assessed via clinical examinations, blood and urine tests, and muscle spectroscopy. Preliminary results after 6 months indicated increased muscle strength and improved subjective function without significant adverse effects, suggesting potential benefits of the treatment combination for SMA patients.
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