Spinal Muscular Atrophy (SMA)

Stem Cell Research: Major Step

The U.S. Food and Drug Administration (FDA) has said yes to a small safety trial of nervous-system stem cells in people with recently sustained injuries to the middle (thoracic) part of the spinal cord, the biopharmaceutical company Geron Corporation of Menlo Park, Calif., announced Jan. 23, 2009.

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.

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