Gene Therapy for Neurological Disorders
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Gene- and cell-based therapies are rapidly evolving to provide disease-altering and potentially curative therapeutic options. Children’s Hospital of Philadelphia (CHOP) has long been on the forefront of the development and application of these therapies for neurological disorders.
engaged in research
active & enrolling
FDA-approved gene therapies for neurological disorders
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Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story
Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.
Programs and services
These clinical programs offer experimental and FDA-approved gene therapies for neurological disorders.
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Our team
The members of our team are global leaders in the development of gene therapies for neurological disorders, bringing visionary research from the lab to the bedside and transforming the future of pediatric medicine.