Summary
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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The Fight Against Rare Diseases
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COVID-19 Impact on Rare Disease Clinical Trials
Rare disease patient organizations are coping with a broad range of other organizational challenges, including reduced funding, canceled fundraising…
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Case Study
PRA Successfully Rescues Phase II Hematology Study
A phase II single-blind, randomized, placebo-controlled trial to study the efficacy and safety of Study Drug administered as adjunctive treatment to…
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Newsletter
RARE DISEASES NEWSLETTER Volume 12, February 2019
This year we are excited to be hosting PRA’s first annual World Rare Disease Day event on February 28th, 2019. The event will be held at the PRA…
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