Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...

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

According to Roche subsidiary Genentech, which announced the label expansion on Monday, TNKase is the first new drug for ...

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 is a small molecule designed to increase the production of working SMN protein by the “backup” SMN2 gene, which in normal conditions can only produce up to 15% of functional SMN protein. A higher ...

This medication helps the body increase SMN protein production by activating a backup gene called SMN2. However, it had never been given to a baby before birth. Since risdiplam is normally approved ...

An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment started in the womb.

Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.