The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

A new electrical stimulation therapy for spinal muscle atrophy (SMA) has shown promise in reactivating motor neurons and ...

The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...

SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein ... leading to muscle weakness over time. Depending on the type of SMA, an ...

“Lack of SMN leads to selective death of spinal motoneurons and progressive muscle atrophy,” the team continued. While there is no cure for SMA ... the motor neurons and engaging the ...

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...

SMA is caused by a mutated or missing survival motor neuron 1 (SMN1) gene, leading to insufficient production of SMN protein and resulting in motor neuron loss, muscle weakness, and paralysis.

pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on ...

Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency ...