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.

faculty engaged in research
active clinical trials

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

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.

  • 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.

Our Team

Next Steps