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

She slept in hospital intensive care units and navigated family outings with a breathing machine – all for her late son, who ...

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 ...

"Our study demonstrated that profound impairments in swallowing are extremely common within the first year of life. This of course has tremendous clinical relevance as it highlights the importance of ...

INDIANANAPOLIS, Indiana -- A mother in Indiana refused to give up on her infant son suffering from a neuromuscular disorder, even when an insurance company and a $2 million price tag stood in her way.

Please provide your email address to receive an email when new articles are posted on . Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic ...

A couple whose son died from a rare genetic condition are encouraging others to take part in screening which they say could ...

This is an open-label study of daily oral risdiplam in infants aged 1 to 42 days with genetically diagnosed SMA. HealthDay News — Infants up to 6 weeks of age with genetically diagnosed spinal ...

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no ...