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.

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

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 ...

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

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 ...

February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists ...

Popular weight-loss therapies like Novo Nordisk's Wegovy and Eli Lilly's Zepbound result in both fat and muscle loss.

Reuters, the news and media division of Thomson Reuters, is the world’s largest multimedia news provider, reaching billions ...

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