Treatment of Spinal Muscular Atrophy

Treatment of Spinal Muscular Atrophy

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.

Read the piece here.

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.

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By |August 11th, 2014|Syndicated Content|

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