Rare Impact Award Given to UPMC Children’s Researcher Dr. Jerry Vockley

Dr. Vockley standing at his lab bench

The National Organization for Rare Disorders (NORD) awarded Dr. Jerry Vockley with the prestigious Rare Impact Award. Rare Impact Awards recognize individuals driving progress for rare diseases. Dr. Vockley serves as director of the Center for Rare Disease Therapy and chief of the Division of Genetic and Genomic Medicine. Dr. Vockley was presented the award in the Scientific […]

Celebrating 10 Years of Nora’s Warriors

A girl on her dad's shoulders facing away from the camera. Their shirts say "Nora's Warriors"

Nora’s Warriors is a Walk for Children’s team that has been walking together since our first Walk for Children’s back in 2016 in honor of their warrior, Nora. Below, Nora’s mom, Andi, tells the story of how Nora’s Warriors came to be. Register for Walk for Children’s today! Our family was introduced to UPMC Children’s […]

Life Without Limits: how a research-based treatment cured Leon of sickle cell disease

Leon and his twin sister, Leigh-Marie, were born in the summer of 2021. When the babies were only about a week old, their parents, Stacy and Leon Sr., received an alarming call from UPMC Children’s Hospital of Pittsburgh. The standard newborn screening test administered to all babies after birth revealed that Leon had sickle cell […]

Liam’s Super Heart: Care from Birth at the Heart Institute

a boy dressed as a superhero pretending to fly while his father holds him up

Baby Liam wasn’t breathing when he was born. He spent the first five nights of his life in the NICU at his local birth hospital, struggling to survive. He was diagnosed with a genetic illness called hypertrophic cardiomyopathy, a condition in which the heart muscle becomes thickened and stiff, making it harder for the heart […]