Raise the Roof: Boosting the Efficacy of a Spinal Muscular Atrophy Therapy

Abstract

Spinal muscular atrophy is the most common genetic killer of infants. A therapy shows promise in the clinic, but there is a potential limit to its efficacy. In this issue of Neuron, d'Ydewalle et al. (2017) devise a new way to make it more effective.