The U.S. Food and Drug Administration has declined to approve a higher-dose version of Biogen's drug for a rare genetic ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...

MIAMI - Celine Domalski was busy riding her unicorn at her Miami Beach condo as she readied to walk in a very important fashion show. "I'm doing a fashion show," Celine told Lisa Petrillo. "Are you ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...

The SMA treatment team also includes rehabilitation professionals such as respiratory, physical, occupational, and speech therapists. Rehabilitation treatment involves care related to positioning, ...

Ten-year-old Sebastian Veiman is racing against time to receive €2.5 million gene therapy that could halt — or even reverse — ...

"The Massachusetts experience demonstrates that SMA NBS is feasible, can be implemented on a population basis, and helps engage infants for early treatment to maximize benefit." --Hale et al., ...

The loss of muscle mass with increased fat mass in patients with SMA suggests a dietary imbalance between energy intake and expenditure. Dietary modifications have shown some promise in managing SMA, ...

Researchers review pulmonary function testing measures that span 4 categories that can assess the respiratory muscle dysfunction that happens in diseases like spinal muscular atrophy (SMA). Muscle ...

Onasemnogene Abeparvovec-Xioi Zolgensma is a therapy medication used for spinal muscular atrophy, a rare disease that affects how cells in the spinal cord tell your muscles how to function. The state ...

A multidisciplinary panel of experts discusses the clinical burden of spinal muscular atrophy on patients, families, and caregivers and considers the importance of patient education and advocacy.

Early diagnosis and treatment of SMA can limit disease progression in children and adults, extending life expectancy and improving QOL. Spinal muscular atrophy (SMA) is a severe genetic condition that ...