Two children with a rare genetic condition will live very different lives because one was tested at birth and the other was ...

The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access ...

Spinal muscular atrophy (SMA) is a genetic disease that causes muscle weakness and wasting, known as atrophy. People with SMA often have difficulties moving, swallowing, sitting up, and sometimes ...

The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...

—The slow progression of SMA in adults and older children complicates evaluation of treatment response to newer therapies. Longitudinal quantitative MRI (qMRI) may give clinicians a better handle on ...

-- Muscular Dystrophy Association Calls FDA Approval of Novartis' Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community Mary Fiance, National Vice ...

On 25 November 2025, Novartis Pharmaceuticals conducted a study is to evaluate the safety, tolerability, and efficacy of the two different treatment combinations of tulmimetostat in participants with ...

SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of ...

"The availability of disease-modifying therapies for patients with other types of SMA suggests that these key disease characteristics in SMA 3 patients should be amenable to therapy." — Barry J. Byrne ...

Spinal muscular atrophy is a genetically inherited disorder that causes muscle weakness. Adults can get spinal muscular atrophy, but it’s rare in adults and progresses slowly. It doesn’t typically ...

Treatment Market is projected to grow at a healthy rate of around 13% by 2026. This growth is primarily driven by the increasing prevalence of SMA, growing awareness of early diagnosis and treatment, ...

Manitoba Premier Wab Kinew says the province is reversing its decision and will ensure a 30-year-old battling a degenerative ...