For the first time ever, doctors have treated a rare genetic disease known as spinal muscular atrophy (SMA) in the womb – and the now 2-year-old child is showing no signs of the disease. SMA is a ...

it would have delayed the approval of Zolgensma, Wilson Bryan, the director of the FDA division that reviewed Novartis’s gene therapy, tells ST AT. The rare disease SMA stems from a mutation in the ...

SMA is a devastating motor neuron disease which affects children. It is caused by an abnormal survival motor neuron (SMN) gene, which leads to a reduction of SMN protein levels. Approximately 50 ...

SMA is caused by autosomal recessive mutations in the survival motor neuron (SMN) gene resulting in reduced SMN protein levels. On the basis of the age of onset and the severity of the disease, four ...