Connecticut prides itself on strong schools, world-class healthcare, and a commitment to caring for its most vulnerable residents. We are a state that values education, innovation, and community. Yet ...
WASHINGTON, Feb. 9, 2026 /PRNewswire/ -- Access to care for Americans living with rare diseases still depends heavily on where they live, according to the National Organization for Rare Disorders ...
The Orphan Drug Act has spurred rare disease research, increasing treatments from 34 to approximately 800, but access remains limited by coverage and payment models. Most rare diseases still lack ...
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Cracking the code of rare diseases
Millions live with rare diseases, yet most remain without a clear diagnosis. Advances in genomics, AI, and multi-omics are transforming how we identify and understand these conditions. Global ...
The new policy acknowledges that biology is personal and that technology now allows medicine to be personal as well. The FDA aims to clear a path for patients (often children) with vexing genetic ...
The National Organization for Rare Disorders (NORD®) and OpenEvidence today announced a new partnership to expand access to trusted, expert-reviewed rare disease information for clinicians, patients ...
The EMA has backed broader pediatric use of Agamree and Crysvita, lowering age thresholds for rare genetic diseases affecting muscle and bone development.
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