World Orphan Drug Congress USA 2017
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Download Pursuing Accelerated Approval for Rare Disease Therapeutics

Retrophin at World Orphan Drug Congress USA 2016

Download Alvin's presentation here for more information on:

The Sparsentan Case Study

  • Sparsentan for the Treatment of Focal Segmental Glomerulosclerosis (FSGS)
  • Sparsentan–Dual Mechanism of Action
  • SparsentanPhase 2 Study –DUET

Accelerated Approval

  • What is accelerated approval?
  • Success stories: Using AA and biomarkers in rare disease drug development
  • What makes for a good biomarker?
  • Making the case for proteinuria as a surrogate endpoint
  • ETA receptor antagonists have shown reduction of proteinuria in various clinical settings
  • Recent advancements on the regulatory front

Find out more about the regulatory pathways in Orphan Drug Research by joining us at the  World Orphan Drug Congress USA 2017 . For example, we have a fantastic Keynote Panel on  Prescription Drug User Fee Act (PDUFA VI) and 21ST Century Cures – recent and pending legislative efforts to make drugs better for patients.

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