Here are the world’s rarest and most bewildering medical conditions, disorders that defy logic, challenge science, and ...

The Orphan Drug Act revolutionized rare disease treatment, yet rising costs and access issues challenge equitable health care solutions.

The crisis facing rare disease and disability families in Connecticut is caused by systemic underinvestment, administrative barriers, and policy choices.

Add Yahoo as a preferred source to see more of our stories on Google. Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested ...

Add Yahoo as a preferred source to see more of our stories on Google. U.S. Secretary of Health and Human Services Robert F. Kennedy Jr. speaks with U.S. Vice President JD Vance (not pictured) during ...

Subscribe to our newsletter for the latest sci-tech news updates. Researchers at the Broad Institute and The Jackson Laboratory have used prime editing, a precise and versatile form of gene editing, ...

Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...

EAU CLAIRE, Wis. (WEAU) - While most families countdown to Christmas, Erin Binder is counting something else: time. Her 14-year-old twin sons, Grady and Jace, both have PKAN, a rare genetic disorder ...

Dec 16 (Reuters) - U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy, to the federal newborn ...

Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...