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 ...
STAT

Prenatal genetic therapy for SMA shows promise in a mouse study

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 ...
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 ...
STAT

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

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 ...
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 ...
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 ...
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 ...

Hundreds of children like Jesy Nelson’s to benefit from ‘life-changing’ drug for muscle-wasting condition

MEDICINES for babies with spinal muscular atrophy will be funded by the NHS for all patients for the first time. Little Mix ...
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 ...