• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials

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.”


To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.