The human body is capable of extraordinary things — but it’s also capable of going spectacularly, bewilderingly wrong in ways most people never imagined. From a man whose gut brews its own alcohol to ...

A rare disease or disorder is defined as affecting fewer than 200,000 Americans each year. There are more than 7,000 different rare disorders. Combined, 1 in 10 Americans has a rare disorder, so the ...

The US Food and Drug Administration (FDA) on Friday published new draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of ...

The World Health Assembly (WHA) Resolution on Rare Diseases was adopted on 24 May, 2025. Persons Living with a Rare Disease continue to face challenges including delays in diagnosis, limited access to ...

Rare diseases, defined as conditions that affect a small percentage of the population, represent a significant challenge in medical research and healthcare. Collectively, these diseases encompass a ...

To promote the development of more treatments for rare diseases, sponsors should leverage data from natural history studies when it is not possible to use data from two adequate and well-controlled ...