Adult-onset spinal muscular atrophy (SMA) is a rare neuromuscular disorder that primarily affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. SMA is part of a group ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs. Previously, ...

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 the motor-neuron condition while in the womb. The child’s mother took the ...

For patients with nonambulatory type 2 or type 3 spinal muscular atrophy, apitegromab, a monoclonal antibody that inhibits myostatin activation, improves motor function versus placebo, according to a ...

Former Little Mix singer Jesy Nelson has been campaigning for screening since her twin girls were diagnosed with the ...

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 ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...