Drug Discovery & Development has included our video in their coverage of a recent Science paper about a new treatment for those with Spinal Muscular Atrophy.
Spinal Muscular Atrophy (SMA) is a debilitating disorder caused by a reduced amount of a protein called SMN. A mutation in the SMN2 gene results in improper splicing of its associated mRNA, leading to deficiency in overall SMN levels. Those affected by SMA suffer from severe motor impairment.
In a pre-clinical study recently published by Science, this new treatment has been shown to effectively increase life span and prevent SMA-related motor dysfunction in mice.
The post Treatment of Spinal Muscular Atrophy appeared first on .