Adult-onset spinal muscular atrophy (SMA) is a rare neuromuscular disorder that primarily affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. SMA is part of a ...

Early intervention in Spinal Muscular Atrophy is crucial for better outcomes, emphasizing the need for timely treatment to ...

Spinal muscular atrophy (SMA) is a genetic disease that ... SMA can strike at any age, from infants through adults. More than 25,000 Americans have the disease. SMA is the No. 1 genetic cause ...

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for ...

Subjects receiving the therapy demonstrated clinically meaningful enhancements in physical and muscle function.

Scientists working at the NIH’s BRAIN Initiative have created new viral vectors that one day could lead to gene therapies for ...

Finally, young children often didn’t open their mouths when asked; the authors point out that if this study is repeated with older children or adults ... of spinal muscular atrophy may differ ...

Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy ... even some adults with ...

Quebec has no age restrictions. A doctor previously told CBC News around 90 per cent of his adult patients with spinal muscular atrophy in the province have benefited from risdiplam.

He hasn't treated Bray, but has worked on a research study of other adults with Type 2 spinal muscular atrophy that showed positive effects from taking risdiplam. Around 90 per cent of his ...