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Pfizer Executive Outlines Vision for Treating Sickle Cell Disease, Other Rare Diseases


Consumers may know Pfizer as the company that manufactures Viagra, but the New York-based pharma giant is investing millions in developing potential treatments and gene therapies for rare disorders ranging from sickle cell disease and hemophilia to ALS and Duchenne muscular dystrophy.

Michael Wajnrajch, MD, is senior medical director for Pfizer’s Rare Disease Group and pediatric endocrinologist on the faculty of New York University. Wajnrajch outlined his company’s vision for gene therapies at the 2nd International Congress on Advanced Treatments in Rare Diseases, held March 4-5 in Vienna, Austria.

“Sometimes we’re doing the right thing for the right reasons, and it’s not about the money,” he told some 100 delegates. “Sickle cell disease predominantly affects patients with very limited ability to pay. We’re not going to make much money on this, but we know it’s an unmet medical need in many parts of the world.”

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