Breaking News
The Economist Who Predicted 2008 Explains China's Endgame
Charlie Kirk SNAPS at Anti White Masked ANTIFA
Episode 488: THE FAUCI FIRESTORM
Discussion with Glen DiesenTop Tech News
Shade-Resistant Solar Cells Retain 97% Efficiency After 2,000 Hours of Testing
20 Ancient Engineering SECRETS
Stonehenge Was Reanalyzed by AI -- And the Findings Are Hard to Explain
After Years Of Delays, Aptera Is Finally Preparing To Build Customer Cars
'When you kill it, it doesn't die': the jellyfish that has cracked the secret of immorta
Archer Aviation debuts Halo autonomous VTOL, Thunder's commercial twin
US Telecoms Slide On Starlink Mobile Threat; Bernstein Sees It As A "Jab, But No Knockout Yet**
Betavolt unveils revolutionary coin-size battery with nickel-63 nuclear isotope...
China claims it has built a 'nuclear drone' that can stay airborne forever
Canada just solved two giant infrastructure problems at exactly the same time.
At $2.1 million, newly approved Novartis gene therapy will be world's most expensive drug
Known as Zolgensma, the gene therapy treats children under 2 years of age with spinal muscular atrophy, an inherited neuromuscular disease that causes progressive loss of muscle function. The most severe form of SMA causes infants to die or rely on permanent breathing support by the age of 2. The disease is caused by a defect in a gene that makes SMN, a protein necessary for the survival of motor neurons. Zolgensma uses a re-engineered virus to deliver a functional copy of the defective gene so that SMN protein can be produced.
Novartis is pricing Zolgensma at $2.125 million, or an annualized cost of $425,000 per year for five years, the company said.
Launching Zolgensma will be a big test for Novartis and CEO Vas Narasimhan, now two years on the job. Shareholders expect the gene therapy to deliver blockbuster sales to justify the $8.7 billion that Novartis spent to acquire it last year.