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MAY 26, 2020

‘An Exciting Time’ to Manage Sickle Cell Disease

Newly approved drugs, gene therapy spark hope

By Frieda Wiley, PharmD
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John J. Strouse, MD, PhD

Sickle cell disease (SCD) affects approximately 100,000 people living in the United States—double the number of people who have cystic fibrosis and hemophilia combined, according to the CDC. For nearly 20 years, the hematologic disorder had only one treatment option: hydroxyurea.

In the last three years, three new drugs have been approved for SCD, offering the community new hope. The FDA approved two of these drugs at the end of