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 ...

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 ...

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 ...

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 ...

Little Mix alum Jesy Nelson is opening up about her 9-month-old twin daughters’ battle with a severe muscle disease. Nelson, ...

- 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 ...

(MEMPHIS, Tenn. – February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children’s Research Hospital led the ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

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

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