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AANS Neurosurgeon | Volume 28, Number 1, 2019

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Preliminary Study Suggests Drug May Help Babies with Spinal Muscular Atrophy

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A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy.

Spinal muscular atrophy (SMA) is an inherited disease that leads to loss of motor function. It is the leading genetic cause of death in infants and toddlers. The disease is caused by reduced levels of the survival motor neuron (SMN) protein. In SMA, the SMN1 gene is mutated or missing. The backup SMN2 gene allows production of some of the necessary protein.

The new drug, called RG7916, is a liquid solution given orally once a day. It is designed to modulate the SMN2 gene splicing to increase SMN protein. 

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