A new approach to spinal muscular atrophy?

Anne Hart

“We’re not suggesting ... that SMA patients should ask their doctors for Riluzole, but we are suggesting that this pathway would be useful for therapeutic development.”

Spinal muscular atrophy is a debilitating neuromuscular disease that in its most severe form is the leading genetic cause of infant death. By experimenting with an ALS drug in two very different animal models, researchers at Brown University and Boston Children’s Hospital have identified a new potential mechanism for developing an SMA treatment.