Gaucher Disease, How a Rare Disease Provides a Window into Common Neurodegenerative Disorders
FDA Approves First Treatment for Weight Management for People with Certain Rare Genetic Conditions
Scientists Use Clues in the Human Genome to Discover New Inflammatory Syndrome
Down Syndrome Research the Intersection of Basic Science and Clinical Cohort Development
NIH-Supported Research Survey to Examine Impact of COVID-19 on Rare Diseases Community
