DANBURY, Conn., Aug. 25, 2025 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®), the leading national nonprofit serving more than 30 million Americans with rare diseases, today ...

Rare diseases affect fewer than 200,000 people in the U.S., approximately 30 million individuals. Sadly, 3 out of 10 children with a rare disease won’t live to see their fifth birthday, yet the path ...

An England-wide study of over 58 million people has identified eight rare diseases that carry significantly increased risks for COVID-19-related mortality in fully vaccinated individuals. This ...

Pangaea Data, a company focused on detecting hard-to-diagnose diseases in patients, is partnering with Alexion, a subsidiary of AstraZeneca focused on treating rare diseases, to co-develop, clinically ...

Crofelemer, a novel plant-based anti-secretory prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines Agency (EMA) for both short bowel syndrome (SBS) and ...

The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.

The FDA has outlined a new review process for drugs and biologics designed to treat ultrarare genetic diseases that would allow a single-arm trial, plus other supportive data, to serve as pivotal ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

A phase 3 trial of AstraZeneca’s $800 million rare disease candidate has met its primary endpoint. The update keeps eneboparatide on track to come to market, but the lack of numbers in the readout ...