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

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

Researchers led by the University of Vienna and Liège University Hospital Center have identified genetic variants associated ...

She slept in hospital intensive care units and navigated family outings with a breathing machine – all for her late son, who ...

A new study published in the Proceedings of the National Academy of Sciences suggests that rare cases of brain inflammation ...

Researchers led by the University of Vienna and Liège University Hospital Centre have identified genetic variants associated with a rare inherited growth disorder in two prehistoric individuals who ...

Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...

Ultra-rare disorders are those affecting fewer than one person in 50,000. Sometimes, indeed, they are unique. And unique ...

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

Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...

Belite Bio (BLTE) stock outlook: tinlarebant clinical data, 2026 filings, key FDA/EU risks, and $165/share valuation. See if ...