At just five months old, Ginny was diagnosed with Spinal Muscular Atrophy (SMA) — a genetic disease that destroys motor ...

Baby Ginny was diagnosed with the most severe form of Spinal Muscular Atrophy and urgently requires donations.

She has a genetic condition that affects motor nerve cells in the spinal cord, causing progressive muscle weakness. Read more ...

Baby Ginny's parents did not ask Singapore to pay for their daughter's treatment. They asked 35,544 strangers. Meanwhile, ...

Zolgensma (onasemnogene abeparvovec-xioi) is a brand-name drug prescribed for spinal muscular atrophy in certain children. Zolgensma comes as a liquid suspension for intravenous (IV) infusion. It’s ...

One month since the landmark approval of Novartis' gene therapy Zolgensma, a handful of top U.S. insurers have set out coverage policies more restrictive than the label granted by the Food and Drug ...

Zolgensma, Novartis’ gene therapy treatment that was approved in May, costs $2.1 million and has often been referred to as the world’s most expensive drug. David Lennon, president of AveXis, the ...

REDONDO BEACH, Calif. -- Ceri and Rory Devine welcomed their first child, Rhys, into the world three months before COVID-19 shut down the country. It wasn't long before the new parents from Redondo ...

When a new drug emerges from research largely funded by grants from charities and government agencies, who gets to set the price? In the U.S., that question seems to have been answered — the drug ...