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

July 1, 2022
Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development
Read More
July 1, 2022
X4 Pharmaceuticals Secures $55 Million Private Placement, Amends Debt Facility
Read More
June 30, 2022
Blueprint Medicines Enters $1.25 Billion Financing Collaborations with Sixth Street and Royalty Pharma
Read More
X