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 ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

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 ...

Megan covers the intersection of science and society. How is biomedical research funded? Who benefits? And how do new technologies reflect and shape our values? You can reach Megan on Signal at ...

What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Nearly six years ago, the Food and Drug ...

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 ...

Itvisma should only be administered intrathecally using a lumbar puncture by health care professionals experienced in performing the procedure. The Food and Drug Administration has approved Itvisma ® ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in uterotreatment of SMA with ...

SMA type 3 is a hereditary disease that affects motor neurons. People may also refer to it as Kugelberg-Welander disease. Symptoms usually begin after 18 months of age. Children with SMA type 3 can ...