Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.

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

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

A new study from the University of Ottawa’s Faculty of Medicine sheds light on a potential breakthrough in treating muscle ...

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

Spinal muscular atrophy is a genetic condition set in motion before birth that causes worsening muscle weakness. There are ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

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 ...

Researchers have developed a regenerative medicine method that successfully grafts myoblasts onto healthy muscle, boosting ...