Month: May 2019

Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) approved by FDA for ATTR amyloidosis cardiomyopathy
This represents the first FDA approved treatment in ATTR-CM for both the wild-type and hereditary forms of ATTR amyloidosis. (more…)
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Patient Perspectives from the FDA Public Meeting: the Impact of Rare Diseases
I recently had the opportunity to attend this public meeting at the FDA to discuss the Patient Perspectives on the Impact of Rare Diseases. This meeting was held as opportunity to bring the voices of patients and caregivers to the...
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