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RDD

Today is Rare Disease Day and we want to recognize the incredible work patient advocacy groups do year-round to raise awareness for the hundreds of millions of people around the world living with — and caring for — rare diseases.

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  • About
    Us
    • Overview
    • Our Leadership
  • Patients
    & Families
    • Overview
    • Lysosomal Disorders
    • Understanding Cystinosis
      • Overview
      • Living with Cystinosis
    • Understanding Gaucher Disease
      • Overview
      • Living With Gaucher Disease (GD1)
      • Living With Gaucher Disease (GD3)
    • Understanding Hunter Syndrome
    • Understanding Pompe Disease
    • Patient Advocacy & Engagement
    • Patient Early Access Policy
    • Statement on Fabry Disease Program
  • Our
    Approach
    • Overview
    • Treating the Whole Body
    • The plato® Platform
    • HSC Gene Therapy
    • Targeting Lysosomal Disorders
  • Our
    Pipeline
    • Overview
  • Work
    with Us
    • Overview
    • Job Openings
  • Our
    Views
    • Overview
    • The Vector Space Blog
    • Ideas and Insights
  • Investors & Media
  • Contact Us