Khymir’s Story: Gene Therapy to Treat Cerebral Adrenoleukodystrophy
Khymir is a 4-year-old boy who was found to have adrenoleukodystrophy (ALD) through Pennsylvania’s newborn screening.
Gene Therapy for Neurological Disorders Patient Stories
Gene therapy has exciting potential for treating neurological disorders. Through cutting-edge research, CHOP is finding new ways to help children with these diseases.