Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...

Globally, Spinal Muscular Atrophy (SMA), a rare genetic neuromuscular disorder, affects approximately 1 in 10,000 live births ...

NBS programs are fairly new in the U.S., so not much is known about variations among them or about provider practice patterns for newborns with SMA. In a survey of providers and state NBS programs, ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

An insurance company has denied newborn twins a one-time genetic treatment that could stop the progression of their spinal muscular atrophy — otherwise they are likely to die before their second ...

Baby Ginny was diagnosed with the most severe form of Spinal Muscular Atrophy and urgently requires donations.

But for Brooklyn, who lives with spinal muscular atrophy, or SMA, a rare genetic condition that affects muscle strength and ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

An experimental treatment that stimulates the spinal cord may help people with a paralyzing genetic disorder called spinal muscular atrophy. An experimental treatment that stimulates the spinal cord ...

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. It leads to ...