Cell and Gene Therapy Patient Stories
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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.
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.
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.
Gene Therapy Treatment for Spinal Muscular Atrophy: Céline’s Story
Céline’s symptoms dramatically improved after she received a new gene therapy treatment for Type 1 spinal muscular atrophy (SMA) at CHOP.
Gene Therapy for Inherited Blindness: Hannah’s Leber Congenital Amaurosis Story
Diagnosed with Leber congenital amaurosis as a baby, Hannah was on a path to going blind until gene therapy at Children’s Hospital restored her sight.
CAR T-Cell Therapy for Relapsed Leukemia: Paulina’s Story
Frantic to save Paulina’s life, her family brought her to the Cancer Center at CHOP where an experimental trial of CAR T-cell therapy cured her cancer.
Relapsed Leukemia: Emily's Story
Emily Whitehead was the first pediatric patient enrolled in an experimental immunotherapy for advanced acute lymphoblastic leukemia.