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 ...

British singer Jesy Nelson recently took to Instagram to share a devastating update about her 8-month-old twin daughters.

The mother of a four-year-old boy with a rare muscle disease says screening newborn babies for the condition could "save ...

Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic infants with spinal muscular atrophy showed significantly improved motor function at 6 ...

SOUTH PLAINFIELD, N.J., May 31, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® ...

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.

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 ...

Lucian Neale was diagnosed with Spinal Muscular Atrophy (SMA) at six weeks old despite showing symptoms in his mother's womb. His mother Samantha Williams, from Crumlin near Pontypool, said she was ...

Jesy Nelson’s fiancé Zion Foster is opening up about their baby daughters' spinal muscular atrophy diagnosis. The former ...