Dive into a community of rare disease patients, caregivers, advocates, and partners from around the world, connected by one passion – improving the lives of people with rare disease.

Community Highlights

October 22, 2021
A Find-and-Replace Approach to Fixing the Mutation Underlying Sickle Cell Disease
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October 22, 2021
Medical Cost for Rare Diseases on Par with Cancer, Heart Failure
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October 22, 2021
Bluebird Withdraws Gene Therapies in Europe as it Winds Down Operations There
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