Gene Therapy for Neurological Disorders
Gene- and cell-based therapies are rapidly evolving to provide disease altering and potentially curative therapeutic options. Children’s Hospital of Philadelphia has long been on the forefront of the development and application of these therapies for neurological disorders.
9
faculty engaged in research
5
active clinical trials
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.
FDA-Approved Gene Therapies for Neurological Disorders
- Zolgensma® for spinal muscular atrophy
- ELEVIDYS for Duchenne muscular dystrophy
- Skysona® for cerebral adrenoleukodystrophy
- Lenmeldy for Metachromatic Leukodystrophy (MLD)
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.
- Duchenne muscular dystrophy (DMD)
- Spinal muscular atrophy (SMA)
- Metachromatic Leukodystrophy (MLD)
- Cerebral adrenoleukodystrophy (CALD)
- Limb-girdle muscular dystrophy (LGMD)
Programs and Services
These clinical programs offer experimental and FDA-approved gene therapies for neurological disorders.
Cell & 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.
- Clinical In Vivo Gene Therapy
- Cell & Gene Therapy Collaborative
- Cell & Gene Therapy Laboratory
- Cell & Gene Therapy Informatics
- Raymond G. Perelman Center for Cellular and Molecular Therapeutics which includes:
- Clinical Vector Core
- Research Vector Core
- Human Pluripotent Stem Cell Core
- Comparative Medicine Services Core
- Engineered Cell Therapy Core