Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

In an exclusive interaction with Professor Philip J Young from the University of Warwick we understood the importance of ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal ...

The FDA approved Itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed SMN1 ...

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 ...