The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).

Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.

A two-and-a-half-year-old girl is defying the odds after becoming the first person to be successfully treated for a rare, ...

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

A new study from the University of Ottawa’s Faculty of Medicine sheds light on a potential breakthrough in treating muscle ...

Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a ...

Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...

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

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.

Mother took Evrysdi for SMA before birth; baby later received Zolgensma. Dr. Richard Finkel at St. Jude led study showing no ...

Earnings included compensation expenses related to equity awards. Excluding these special items, adjusted loss per share was 43 cents against a gain of 12 cents per share in the year-ago quarter.