The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no ...

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal stimulation. Spinal stimulation restored muscles wasted by rare genetic ...

An experimental treatment that stimulates the spinal cord may help people with a paralyzing genetic disorder called spinal muscular atrophy. An experimental treatment that stimulates the spinal cord ...

Edward was diagnosed with spinal muscular atrophy (SMA) when he was just 2 months old, his mother told the BBC He was given the gene therapy, Zolgensma, back in 2021, with the drug costing around $2.4 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...

Patients with SMA often suffer from impaired bulbar function, which negatively affects quality of life and can be difficult to treat effectively. A German study prospectively followed children with ...

Daily electrical stimulation of certain nerves in the spinal cord appeared to help three people with spinal muscular atrophy (SMA), an inherited disorder that causes muscles to waste away. The ...