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

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

The classification of spinal muscular atrophy (SMA) subtypes, the influence of SMN2 gene copies on disease severity, treatment effects on disease progression, age-related differences, long-term ...

In its most severe form, as in the case of this child, individuals lack both copies of the SMN1 gene, and have only one or two copies of a neighbouring gene, SMN2, that partially compensates for ...

Bio-Techne's Asuragen brand inks a partnership with Oxford Nanopore Technologies to launch research-use-only AmplideX Nanopore Carrier Plus Kit.

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

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 ...

who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively.