“BelleDozer” Triumphs over Thalassemia

Belle was adopted from China when she was 19 months old. When her parents first met her, they knew she was special. They also knew Belle had potentially devastating blood disorder called thalassemia, which would require a lot of medical care.

How Donors are Fueling Ewing Sarcoma Research

For Kelly Bailey, MD, PhD, Ewing sarcoma is more than a rare and difficult-to-treat bone tumor that predominantly affects teenagers. It’s the focal point of her research and the primary reason she practices medicine.

Meet Rosie

Shortly after Rosie was born, physicians diagnosed her with Maple Syrup Urine Disease (MSUD), a rare, life-threatening genetic disorder in which the body is unable to process certain proteins.

Type 1 Diabetes: Closing in on a Cure

A UPMC Children’s physician-researcher and his team are closer than ever to discovering a new treatment for over 200,000 children, adolescents, and adults living with diabetes in the United States.