Spinal Muscular Atrophy (SMA)

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.

SMA Research: Back to the Beginning

In a development that could lead to better screening of drugs for spinal muscular atrophy (SMA), skin cells from a child with SMA1 have been "reprogrammed" back to a stemlike state and then coaxed to develop into SMA-affected motor neurons, the nerve cells that normally control muscle movement but malfunction and die in this disease.