Division of Hematology Patient Stories

Every person on your child's team has the same goal: to see your child thrive. We provide medical care, emotional counseling and much more.

How we serve you

215-590-3535

Patient story

Gene Therapy for Sickle Cell Disease: Segun’s Story

An FDA-approved gene therapy eliminates acute pain episodes for people with sickle cell disease and transforms their life expectancy.

Patient story

Gene Therapy for Sickle Cell Disease: Marie-Chantal’s Story

An FDA-approved gene therapy is giving people with sickle cell disease a future they didn’t think they’d have.

Patient story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

Bill’s hemophilia caused several serious health crises. After nearly 60 years of continual treatments, a gene therapy transformed his life.

Patient story

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.

Patient story

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.

Patient story

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.

Patient story

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.

Patient story

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.

Patient story

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.

Patient story

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.

Find a research study

Innovation and your child

Find research studies

Division of Hematology Patient Stories

Gene Therapy for Sickle Cell Disease: Segun’s Story

Gene Therapy for Sickle Cell Disease: Marie-Chantal’s Story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

Experimental Gene Therapy for Hemophilia B: Jay’s Story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

Shwachman-Diamond Syndrome: Elizabeth’s Story

Bone Marrow Transplant for Sickle Cell Disease: Asher’s Story

Bone Marrow Transplant for Sickle Cell Disease: Dallas’ Story

Experimental Gene Therapy for Beta Thalassemia: Aliya’s Story

Beta Thalassemia Major: Mia’s Story