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 ...

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.

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

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 ...

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

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 ...

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 ...

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

Annji Pharmaceutical Co. Ltd.’s rosolutamide (AJ-201, JM-17) achieved positive results in a phase I/II trial in adults with spinal and bulbar muscular atrophy, also known as Kennedy’s disease.