Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to ...

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 ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

SMA pathology affects a wider range of cells, including astrocytes and microglia, beyond alpha motor neurons. Advanced techniques revealed significant ventral horn neuron loss and abnormal morphology ...

"Our study demonstrated that profound impairments in swallowing are extremely common within the first year of life. This of course has tremendous clinical relevance as it highlights the importance of ...

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and ...

INDIANANAPOLIS, Indiana -- A mother in Indiana refused to give up on her infant son suffering from a neuromuscular disorder, even when an insurance company and a $2 million price tag stood in her way.

A new newborn screening test recently saved the life of a little girl with spinal muscular atrophy, a rare neuromuscular disease. Thanks to this test, now widely used in France, other children could ...