AAN 2018: Investigational drug offers hope for spinal muscular atrophy

Written by Sharon Salt, Editor

A preliminary, open-label study presented at the American Academy of Neurology’s 70th Annual Meeting (April 21–27, CA, USA) has revealed that an investigational drug may help increase protein levels in babies with spinal muscular atrophy (SMA). SMA is the leading genetic cause of death in infants and toddlers and is caused by a reduction in SMN protein levels. In SMA, the SMN1 gene is either mutated or missing. To override this, the SMN2 gene can act as a backup to allow production of some of the necessary protein. The novel drug – termed RG7916 – is a liquid solution given...

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