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 ...

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 ...

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

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 ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...

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 ...

Patients with SMA often suffer from impaired bulbar function, which negatively affects quality of life and can be difficult to treat effectively. A German study prospectively followed children with ...

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

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.