Opportunities in the SMA market include leveraging the launch of new myostatin inhibitors, expanding Zolgensma formulations to increase patient eligibility, and capitalizing on cheap nusinersen ...

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

But while Marley, who was diagnosed at five months, cannot walk or talk and needs help to breathe, Meadow is meeting all the milestones for her age after she was tested at birth and received ...

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

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 ...

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 ...

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.

Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of ...

The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed by Novartis, a major step forward for ...

—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 ...

Jeremy Bray, 29, lives with Spinal Muscular Atrophy, a neurodegenerative disorder that causes the loss of motor neurons and ...