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For the first 30 years of her life, Aliya needed monthly blood transfusions for her inherited blood disorder. A gene therapy changed everything.
Céline’s symptoms dramatically improved after she received a new gene therapy treatment for Type 1 spinal muscular atrophy (SMA) at CHOP.
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.
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.
Emily Whitehead was the first pediatric patient enrolled in an experimental immunotherapy for advanced acute lymphoblastic leukemia.