The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

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 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 ...

Please provide your email address to receive an email when new articles are posted on . Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic ...

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 ...

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 multicenter pilot study concluded that dual therapy in spinal muscular atrophy (SMA) does not provide further benefits to infants at risk for SMA type 1. In the last decade, the approval and ...

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 ...