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 ...

Doctors in the US have treated a fetus with a rapidly progressive congenital disease while in the uterus for the very first ...

A baby diagnosed with spinal muscular atrophy (SMA) was successfully treated in the womb for the first time. Now over two years old, she shows no signs of the disease. This milestone suggests that ...

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 ...

The three diagnostic tests for identifying genetic mutations associated with SMA met new European Union regulatory ...