Drug May Help Babies with Spinal Muscular Atrophy

18th April, 2018

20 April 2018, News, Newsletter

[Source: Medical X-Press]

A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy. The open-label study is released today and will be presented at the American Academy of Neurology’s 70th Annual Meeting in Los Angeles, April 21 to 27, 2018.

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.

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