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Menu
  • About
  • Cell & Gene Medicine
    • Understanding Cell & Gene Medicine
    • Cell Therapy
    • Gene Medicine & Gene Therapy
    • Gene Modified Cell Therapy
    • Gene Editing
    • Gene Silencing
    • Tissue Engineering
    • Regenerative Medicine
    • Approved Cell & Gene Therapies
    • Making Informed Decisions
  • Access Info by State
    • About STRiDE
  • Speaking of Genes
    • First 20 Glossary
    • Next 20 Words for Review
    • Nomenclature Summit Report July 2020 (PDF)
    • Beginner Zone
  • Patient Organization Toolkit
    • Understanding Cell and Gene Medicine (Slide Deck)
    • Understanding Cell and Gene Medicine (Guidance)
    • For Patient Organization Leaders
  • Resources
    • Available Treatments
    • Clinical Trial FAQs
    • Info on Specific Diseases
    • Paying for Therapy
    • Support Organizations
    • Video FAQ
  • News & Events
    • News
    • Events
  • Contact Us

Disease Information

duchenne muscular dystrophy
epidermolysis bullosa
hemophilia a
hemophilia b
huntington's disease
oncoloy: multiple myeloma
lysosomal storage disease
sickle cell disease
spinal muscular atrophy

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