First Round Abstract Submission is Open Now...

Rare Disease & Orphan Drugs

Conditions known as rare diseases only impact a tiny portion of the population. Medication created especially to address these uncommon illnesses is known as an orphan medicine. Since most uncommon diseases have a hereditary basis, they usually persist for the entirety of a person's life, even in cases when symptoms do not show up right away.

  • Orphan Drug Development
  • Patient-Centered Research & Advocacy
  • Real-World Evidence
  • Pediatric Rare Diseases
  • Early Detection
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