MedPage Today

Newborn Screening for Spinal Muscular Atrophy: Early Diagnosis and Treatment

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 ...
MedPage Today

Newborn Screening for Spinal Muscular Atrophy: How Is the U.S. Doing?

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

New Gene Therapy Possible For Infants With Spinal Muscular Atrophy

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 ...
The American Journal of Managed Care

Overview of Spinal Muscular Atrophy

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 ...
News Medical

First prenatal treatment for spinal muscular atrophy shows promising results

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 ...
Medical Xpress

What you should know about spinal muscular atrophy

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 ...
STAT

Prenatal genetic therapy for SMA shows promise in a mouse study

Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT in 2021 after covering health and science at WIRED. You can reach ...
WebMD

Evrysdi for Spinal Muscular Atrophy: What You Need to Know

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 ...
Emirates News Agency on MSN

Tawam Hospital administers groundbreaking gene therapy for child with spinal muscular atrophy

Al AIN, 11th May, 2026 (WAM) -- SEHA, a subsidiary of PureHealth, has revealed a significant medical milestone achieved at Tawam Hospital with the successful administration of gene therapy for Spinal ...
AOL

What is SMA? The rare genetic condition that Jesy Nelson’s twin daughters have

Former Little Mix star Jesy Nelson has revealed her twin daughters, Ocean Jade and Story Monroe Nelson-Foster, have been diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic condition ...
STAT

Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?

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 ...