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.
Cell and Gene Therapy Innovations Patient Stories
Building on our proven track record in cell and gene therapy, CHOP is committed to discovering the next life-changing treatments for the most serious diseases.
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 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.