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Cure Rare Disease is a nonprofit biotechnology company whose mission is to enable and finance the development of life-saving genetic medicines for rare and ultra-rare patient populations previously deemed too rare to treat. To realize this, our unique ecosystem facilitates collaboration between world-renowned researchers and clinicians, policy experts and our generous donors. Together, we are fundamentally changing the rare and ultra-rare disease experience and burden for millions of people around the world with our discoveries and methods—while bringing hope to patients waiting for a cure.

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drug development, research, advocacy, education, pharmaceuticals, precision therapy, gene editing, crispr, nonprofit, fundraising, accessibility, duchenne, rare disease

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18
2017
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