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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.”

Apr 4, 2019
clinical trials and research

Johns Hopkins researchers offer new protocol to potentially cure sickle cell disease

There's new hope for patients who have blood diseases. Johns Hopkins Kimmel Cancer Center researchers say a new protocol offers the possibility of a cure. One patient who tried the treatment says it didn't just change her life, it saved it.

For most of her life, Simone Day had a very different experience at the hospital than the one she's having now. For Day, sickle cell meant acute pain crises. She'd be in the hospital for weeks. "I was essentially OK with dying at that point," Day said. It's different now, thanks to a bone marrow transplant in 2017.

The new protocol doesn't only mean using what they call "half matched" donors, like Day's mother, but also lowering the dosages of chemotherapy to make it safer on the patient.


Mar 20, 2019