Collectively, rare diseases represent a large medical challenge. While most have no cure, the appropriate treatment can greatly improve quality of life and extend life expectancy. Impressive progress has been made, so we must not give up the fight. A disease might be rare, but hope should not be.
Patient-Centric Trial Development Toolkit
Learn more about our practical tools for sponsors, participants and champions of patient-centric trial development and how you can start using them…
Patient-Centric Protocol Risk Assessment Tool
Expert-validated tool that enables sponsors to identify potential trial efficiency risks driven by participation burden in any draft protocol
Studying Rare Diseases Helps Advance Common Disease Treatment
Many rare diseases have unexpected connections with common diseases. These relationships can lead us to treatments not only for these rare diseases,…