A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...

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

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 ...

In general, each corresponds to the copy number of the SMN2 gene, which produces a small fraction (~10%) of functional SMN protein, essential to the survival of motor neurons, compared with SMN1.

or patients with 5q SMA with a bi-allelic mutation in SMN1 and up to three copies of the SMN2 gene. Zolgensma is the world’s most expensive drug, with a price tag of more than $2.1 million per ...

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.