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25.05.2019 01:46:00

Approval of a Game-Changing Gene Therapy for SMA Delivers a Win for Novartis and Regenxbio

There are over 6,000 genetic disorders, and most don't have any regulatory-approved treatment options. That's discouraging, but there's reason for hope; gene therapies are in development that aim to address the root cause of genetic diseases. On Friday, the Food and Drug Administration (FDA) gave a green light to Novartis' (NYSE: NVS) Zolgensma, a revolutionary gene therapy for spinal muscular atrophy (SMA).Zolgensma's $425,000-per-year price tag suggests it will be a blockbuster for Novartis; if it is, then Regenxbio (NASDAQ: RGNX) could pocket nine figures per year in royalties. Here's what you need to know about this important approval.IMAGE SOURCE: GETTY IMAGES.Continue reading
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