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

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

-- 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 ...

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...

Treatment Market is projected to grow at a healthy rate of around 13% by 2026. This growth is primarily driven by the ...

Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor ...

Leptin merely correlated with body mass index, the study found. A new report on patients with types II and III spinal muscular atrophy (SMA) has found that levels of the peptide hormone leptin are ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

"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 ...