Division of Hematology Patient Stories
1 - 10 of 13
Experimental Gene Therapy for Hemophilia B: Jay’s Story

For 50-plus years, Jay dealt with the frequent treatments needed for his hemophilia. A gene therapy put an end to the treatments and to the worry the disease caused him.
Experimental Gene Therapy for Hemophilia B: Bill’s Story

For nearly 50 years, Bill needed frequent treatment for hemophilia — often every few days. A gene therapy changed everything.
Shwachman-Diamond Syndrome: Elizabeth’s Story

Born with a rare condition affecting her bone marrow, 2-year-old Elizabeth has large care teams at CHOP and her local hospital who coordinate to keep her growing and happy.
Bone Marrow Transplant for Sickle Cell Disease: Asher’s Story

For years, sickle cell disease caused frequent pain crises and hospitalizations for Asher. A curative therapy has eliminated those harrowing experiences.
Bone Marrow Transplant for Sickle Cell Disease: Dallas’ Story

Sickle cell disease brings constant fear of life-threatening complications. A successful bone marrow transplant ended those fears for Dallas and her family.
Experimental Gene Therapy for Beta Thalassemia: Aliya’s Story

For the first 30 years of her life, Aliya needed monthly blood transfusions for her inherited blood disorder. A gene therapy changed everything.
Beta Thalassemia Major: Mia’s Story

From the time she was just a few months old, Mia needed blood transfusions due to her rare blood disease. A bone marrow transplant changed everything.
Cyclic Neutropenia: Ella’s Story

For years, Ella’s health seemed fragile, culminating in a life-threatening infection. She now needs injections to control her rare blood disorder.
Shwachman-Diamond Syndrome: Joyce’s Story

A very rare condition makes Joyce vulnerable to infections and long-term complications, but she remains wonderfully independent and adventurous.
Beta Thalassemia Major: Nicholas's Story

Beta thalassemia major forced Nicholas to undergo regular blood transfusions until he received a lifesaving bone marrow transplant.