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 ...

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

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 ...

Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families.

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 ...

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 ...

MRI imaging scans can track muscle changes in SMA children given Spinraza, a study found, with better results seen with early ...

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 ...