In a groundbreaking medical achievement, doctors have successfully treated a life-threatening motor neuron disease in a baby ...

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

A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

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

This rare genetic disorder was just treated in the womb for the first time ever - Without treatment, those with the more ...

Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy: Basel Friday, February 14, 2025, 13:00 Hrs [IST] Roche, announced that the US Food ...

Over the past nine years, patients with spinal muscular atrophy have seen the number of FDA-approved treatment options for ...

The Evrysdi oral solution will remain available for those ... It affects approximately one in 10,000 babies and is the ...

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

The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water ...