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

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 ...

Evrysdi is a non-invasive, disease-modifying option that can be swallowed whole or dispersed in water for patients with ...

A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...

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

Roche has announced that a tablet formulation of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) has been ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in uterotreatment of SMA with ...

Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.

Novartis NVS and AbbVie ABBV announced small M&A deals. The FDA approved the expanded use of Pfizer’s PFE cancer drug ...

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

Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular ...

Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease ...