Welcome to the thirteenth edition of the rare diseases newsletter. This quarterly publication strives to keep you up to date on PRA's Center for Rare Diseases achievements and new initiatives.
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Laura Iliescu, Manager of Patient Advocacy & Engagement, 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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Decoding FDA Gene Therapy Guidances Part Two: Rare Disease
In Part Two of Decoding FDA Gene Therapy Guidances, we discuss three separate guidances that provide further insight into how sponsors can safely and…
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Finding a Cure for Myasthenia Gravis
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