Valproic Acid in SMA

Preliminary results in the phase 2 study of valproic acid and carnitine in children with types 2 and 3 spinal muscular atrophy (SMA) suggest the treatment may have improved function in children younger than age 3 who were not walking at study entry. However, there was no benefit of the drug treatment when a larger group that included children ages 2 to 8 who were not walking was compared with a placebo (inactive substance) group. See Valproic Acid and Carnitine in Patients with SMA: Phase 2 Study.

Troy Lino
Valproic acid may help cells produce more SMN, which is deficient in SMA. Early trial results have been disappointing, but studies continue.

The children who received valproic acid and carnitine gained more fat mass than those in the placebo group, which appears to have interfered with motor function.

Study continues in walking and standing children

Results in SMA-affected children 3 to 17 years old who could stand or walk at study entry expected by the end of 2009.

Study recruitment is complete.

Drug also being studied in infants, adults with SMA

A phase 1-2 trial of valproic acid and carnitine in infants 2 weeks to 9 months old with type 1 SMA (Valproic Acid and Carnitine in Infants with SMA1) remains open. This trial is also known as CARNIVAL Type 1.

Also open is a trial of valproic acid in walking adults with type 3 SMA (Valproic Acid in Ambulant Adults). This trial is also known as VALIANT SMA.

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