Pfizer's Vyndaqel (tafamidis) has been approved in the EU as a treatment for transthyretin familial amyloid polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.
Mutations of the transthyretin (TTR) gene can result in the production of unstable TTR proteins which can accumulate as amyloid...
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