The FDA has approved an adeno-associated virus 9 gene replacement therapy to treat individuals aged 2 years and older with ...

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 ...

Marley was given Zolgensma, a life-changing gene therapy drug, at 16 months. According to SMA UK, the drug delivers a healthy copy of the affected gene to the body, but timing is critical because ...

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

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

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 latest announcement is out from Scholar Rock Holding ( (SRRK) ).

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.

-- 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 ...

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 ...

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