Early diagnosis and treatment of SMA can limit disease progression in children and adults, extending life expectancy and improving QOL. Spinal muscular atrophy (SMA) is a severe genetic condition that ...

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 ...

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 ...

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

PITTSBURGH (KDKA) -- August is SMA Awareness Month. SMA stands for Spinal Muscular Atrophy. It is a rare neuromuscular disorder that starts in the central nervous system and affects all of the muscles ...

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

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 ...