Experts at Cincinnati Children's are playing major roles in improving outcomes for people with sickle cell anemia.
In Africa, where sickle cell kills many children before age 5, a four-nation clinical trial led by Dr. Russell Ware and colleagues reports that the inexpensive drug hydroxyurea is safe and effective for people there. Read the NEJM study.
Meanwhile, the first two people to join a clinical trial of a new gene therapy developed by Dr. Punam Malik are showing significant benefit at the one-year mark. Learn more.
Cincinnati Children's also recently agreed to work with Swiss-based Roivant Sciences to form Aruvant Sciences to continue developing the new gene therapy. Learn more about the company.
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