Doctors in the US have treated a fetus with a rapidly progressive congenital disease while in the uterus for the very first ...

DAVENPORT, Fla. - The Sunshine Foundation is set to create a magical moment for Julianna Rodriguez, a young girl battling ...

Spinraza treatment may help restore lipid balance to the cerebrospinal fluid (CSF) of children with SMA, according to a small ...

Vriddhi, a seven-month-old girl, is battling spinal muscular atrophy type 1, a rare progressive disease that will cause her muscles to weaken over time. The only hope for her survival is a gene ...

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The scientists treated spinal muscular atrophy, a debilitating genetic condition which affects motor neurons that control ...

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

The revolutionary drug, called risdiplam, was taken by the mother during her 32nd week of pregnancy and is now part of the ...

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

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

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 ...

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