[Comment] Genetic therapies for spinal muscular atrophy type 1

Spinal muscular atrophy (SMA) type 1 —the leading genetic cause of infant mortality—results from an absence of functional copies of the SMN1 gene, which encodes survival motor neuron protein (SMN). The course of this disease might soon change, however, as outlined by two publications on successful trials with SMA therapies.1,2
Source: Lancet Neurology - Category: Neurology Authors: Tags: Comment Source Type: research