Novartis gene therapy approved, but will come at cost of more than $2 million

cafead

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  • cafead   May 24, 2019 at 11:52: PM
via Babies born with a severe form of a rare genetic condition known as spinal muscular atrophy almost always die before their second birthday.

A gene therapy approved Friday by the Food and Drug Administration promises to change that, offering a potential one-time fix for the genetic deficiency behind the neuromuscular disease.

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