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Untreated, children with SMA type 2 will never be able to walk. After receiving a one-time dose of gene therapy, William has gained strength and hit milestones his family previously didn't think possible.
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.
With a breakthrough drug for spinal muscular atrophy treatment and ongoing therapy at Children’s Hospital of Philadelphia, Claire is reaching new milestones.