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 ...

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

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

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 ...

- 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 ...

Jesy Nelson of Little Mix fame has shared the news that her twin girls have been diagnosed with SMA Type 1, a genetic disorder that is primarily diagnosed in children. The singer shared an emotional ...

Jesy Nelson has bravely shared a devastating diagnosis for her twin babies - that they will never walk. The Little Mix star revealed her baby twins have been diagnosed with the incurable genetic ...

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 ...