We’ve been working on it for a long time so to have it read out positively, it’s exciting but also moving. It’s a really underserved patient population and a uniformly fatal disease.
—Brenda Cooperstone, Pfizer’s chief development officer for rare disease, talking about late-stage clinical trial success of tafamidis, the firm’s experimental treatment for transthyretic cardiomyopathy (April 2)
We developed a way to computationally find matches between rare disease protein structures and functions and existing drug interactions that can help treat patients with some of these orphan diseases.
—Louisiana State University PhD student Misagh Naderi, in a press release announcing her recently published paper on drug repurposing in npj Systems Biology and Applications (March 13)
LUIZA AUGUSTO1. ...