Blood and Marrow Transplant Program Patient Stories
1 - 10 of 22
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.
X-linked Lymphoproliferative Disease 1: Rafi’s Story
Genetic sequencing revealed that Rafi had an extremely rare, often fatal disease — and the discovery was amazingly made before he had serious symptoms.
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.
Bone Marrow Transplant to Treat Hurler Syndrome: Josie’s Story
18-month-old Josie received a stem cell transplant to stop a rare and devastating genetic condition in its tracks.
Bone Marrow Transplant for Hurler Syndrome: Ethan’s Story
Diagnosed before his first birthday with a rare disease that causes ongoing damage to cells and organs, Ethan quickly received the only treatment that prevents further disease.
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.
Endocrine Late Effects After Cancer Treatment: Raine’s Story
Treatment for childhood leukemia has had several long-term effects on Raine’s health, but this successful college graduate is pursuing her dream.
Beta Thalassemia Major: Nicholas's Story
Beta thalassemia major forced Nicholas to undergo regular blood transfusions until he received a lifesaving bone marrow transplant.
Medulloblastoma and Kawasaki Disease: Gabby’s Story
"CHOP is so great because they support the whole family," says Gabby's dad, Paul.