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 ...

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 ...

(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 ...

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 ...

HONOLULU (KITV4) -- Oahu student and artist Janelle Fiesta, 26, has been living with spinal muscular atrophy (SMA), a rare, progressive neuromuscular disease, her entire life. She is the first person ...

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 ...

SAN DIEGO (KGTV) — Alan Alvarez, a former San Diego police officer that shared his story of battling a rare disease with ABC 10News over the last few years, has passed away. Alvarez suffered from ...