Gene Therapy for Neurological Disorders

Gene Therapy for Neurological Disorders

At Children’s Hospital of Philadelphia, groundbreaking science meets compassionate care for children with neurological disorders.

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.

9
faculty

engaged in research
5
clinical trials

active & enrolling

FDA-approved gene therapies for neurological disorders

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.

Conditions we treat

At CHOP, we are always looking for new and innovative ways to treat all neurological and muscular disorders. Currently, there are four of these such diseases that we can treat with experimental or FDA-approved targeted gene therapies.

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Programs and services

These clinical programs offer experimental and FDA-approved gene therapies for neurological disorders.

Cell and gene therapy supportive programs

The path from an idea in the lab to a clinical trial and finally to an FDA-approved therapy winds through these support programs. It’s with the expertise of these programs that discoveries are ushered through the complicated but critical steps toward safe and effective treatments.

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Lab worker sitting at microscope and smiling

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.