"Monumental" is how Ashley E. Webb, MD Assistant Professor, Department of Pediatrics, Division of Child & Adolescent Neurology Neuromuscular Program Director, UTHealth, University of Texas Health ...
It took some time before Lauren Benz could bring herself to visit a playground with her young son Leonardo. Because of physical limitations resulting from spinal muscular atrophy, watching other ...
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 ...
"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...
Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...
FRESNO, Calif. -- Richard and Kaylee Lazaro had just welcomed twins Rykker and Bekkem, but Rykker was rushed to the NICU and later diagnosed with Spinal Muscular Atrophy (SMA). He was able to receive ...
MADERA COUNTY, Calif. -- Like most 1-year-olds, Damaris Perez spends a lot of time playing in her crib. The average person wouldn't know this baby has limited time left on earth. "It's been different, ...
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 ...
A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...
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