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 ...

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.

The Pitt study was conducted as part of a pilot clinical trial that enrolled three adults with milder ... Chen, of the Spinal Muscular Atrophy Foundation. This research was supported by an ...

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 ...

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

Scholar Rock contends its experimental drug for spinal muscular atrophy can achieve what none ... benefits of apitegromab to children and adults with SMA in the U.S., Europe, and around the ...

Spinraza (nusinersen) treatment helped school-age children with SMA be more independent in tasks involving their arms and ...

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.

Spinal muscular atrophy (SMA) is a rare genetic disorder ... About 2 percent of American adults are carriers of SMA. If both parents are carriers, there’s a 1 in 4 chance that their child ...