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

September 24, 2021
Creating a Playbook for Bespoke Gene Therapies
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September 24, 2021
Long-Term Treatment with Crysvita Reduces the Burden of Disease in Adults With XLH
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September 24, 2021
AGTC Reports Serious Adverse Events in Three Patients
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