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

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

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 ...

With a tank of zebrafish, Queensland researcher Jean Giacomotto was able to test a never-before seen genetic variant ...

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...

Children with spinal muscular atrophy (SMA), a rare autosomal recessive disease that results in progressive muscle weakness and atrophy, can suffer the concerning consequences of dysphagia, which ...

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

A mother whose son was diagnosed with spinal muscular atrophy (SMA) when he was ten days old has urged singer Jesy Nelson to "take all the help she can get" for the condition. The former Little Mix ...

Jesy Nelson of Little Mix fame has shared the news that her twin girls have been diagnosed with SMA Type 1, a genetic ...

- Taldefgrobep alfa, a myostatin-targeting biologic investigational agent, in Phase 3 development to increase muscle mass for Spinal Muscular Atrophy patients now granted Fast Track in addition to ...

Footballer Ezekiel Otuoma and his wife Rachael Otuoma. Otuoma died after a long battle with Motor Neurone Disease (MND). [File, Standard] Former AFC Leopards winger Ezekiel Otuoma passed away at the ...

Amsterdam, February 14, 2023 – A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been ...