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.
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Partnering with Patients to Develop Meaningful Endpoints – Practical Considerations
Selection of appropriate endpoints for rare disease clinical studies can be challenging but is critical to assessing the efficacy of new medicines.…
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Amy Raymond, PhD, Director of Therapeutic Expertise, Center for Rare Diseases
We measure our HQ with actions, experiences, and intentions. Nowhere is that more evident that in the stories of our subject matter experts. We want…
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Guiding Principles for Interactions with Rare Disease Patient Advocacy Organizations
PRA acknowledges and thanks the members of the Rare Disease Advisory Committee (RDAC) who provided their valuable contributions to the development of…
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