SMA Type 1 is the leading genetic cause of infant mortality around the world. It results from a defect in the SMN1 gene, and about one in 50 Americans are genetic carriers. In the absence of a ...

Testing SMA patient DNA with advanced methods like HapSMA can reveal genetic changes that affect how severe the disease is, ...

The iPS-SMA neurons were deficient in SMN protein, but began to express SMN in response to drug treatment, suggesting that the iPS-derived neurons could be used in drug development screens.