Neurological Gene Therapies Patient Stories
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Khymir’s Story: Gene Therapy to Treat Cerebral Adrenoleukodystrophy
![patient-story-khymir-martin-canonical.jpg](https://www.chop.edu/sites/default/files/styles/teaser/public/patient-story-khymir-martin-canonical.jpg?itok=qLLWHKZ9)
Khymir is a 4-year-old boy who was found to have adrenoleukodystrophy (ALD) through Pennsylvania’s newborn screening.
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.