Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.

Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.

The midwestern state has quietly laid the groundwork for a biotech hub.

The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.

The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.

Novartis, which bought the biotech firm in 2018, announced that the company’s top scientists have left, and news reports say it’s because they were involved in using manipulated data to get the gene therapy Zolgensma approved.

The agency is now evaluating the implications of the corrupted data behind Zolgensma and whether to “take action” against the pharmaceutical company.

At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.