View highlights from our research programs, community and team in 2020.
This year will undoubtedly be remembered as a dividing line in many of our lives. Life pre-pandemic and life post-pandemic will forever be different chapters of our stories.
But throughout 2020, you — our community — were a constant source of inspiration. You give us a deep sense of purpose and urgency in our mission to unlock new treatments for people with rare disease. We made exciting progress toward that goal in 2020.

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