FDA Approval, Gene Editing and Gene Silencing

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

JULY 13, 2021

At the moment, the only FDA-approved treatments for ATTR cardiomyopathy are Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), which were given a green light by the agency two years ago. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body.

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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

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