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      • Adrenal Insufficiency
      • Congenital Adrenal Hyperplasia
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      • Cystic Fibrosis
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Skip to content
  • About Us
    • About EffRx
    • Business Strategy
    • Management Team
    • Board of Directors
    • Investors
  • Therapeutic Areas
    • Adrenal Insufficiency
      • Adrenal Insufficiency
      • Congenital Adrenal Hyperplasia
    • Cystic Fibrosis
      • Cystic Fibrosis
      • Unmet Medical Need
    • Lysosomal Storage Disorders
      • Gaucher Disease
      • Niemann-Pick Disease Type C
    • Osteoporosis
      • What is Osteoporosis?
      • Unmet Medical Need
      • The Adherence Gap
  • Products & Pipeline
    • Products
      • Binosto®
      • Alkindi®
      • Miglustat
    • Pipeline
      • Pipeline Overview
      • Bronchitol®
      • Efmody®
  • Business Development
    • Rare Diseases
      • Rare Diseases in Europe
      • Orphan Medicines in Europe
    • European Market
    • Affiliations
    • World Orphan Drug Alliance (WODA)
  • Newsroom
    • Press Releases
    • News
  • Locations

Therapeutic Areas

Rare Diseases in Europe

Orphan Medicines in Europe

The Adherence Gap

Unmet Medical Need

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