Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?

The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for ...

Empowering patients: Often, rare disease patients endure a delayed and psychologically difficult diagnosis process, involving a lack of information, stigma and social isolation, ...

Rare diseases present a perfect paradox for human diagnosticians. Their unfamiliarity makes them difficult to recognize, yet many leave subtle patterns of symptoms, lab results, ...

Bruce Leuchter, CEO, Neurvati, discusses the vital role of public participation in healthcare policy, highlighting how ...

Senators made the right decision for America's most vulnerable patients ― now, the House must follow suit. The ORPHAN Cures ...

Expansion of the Medicare drug price negotiation rare disease drug carve-out would unnecessarily limit the number of drugs ...

Rare Disease Day is observed every year on the last day of February, the rarest day of the year In India, a disease is considered rare if it affects fewer than 100 people per 1,00,000 individuals ...

In her spare time, Oxana volunteers at the International Rare Disease Research Consortium (IRDiRC), where she is co-chair of the Regulatory Scientific Committee (RSC). In this interview, Oxana ...

Innovation and Progress in Rare Disease Trials. Despite these hurdles, rare disease clinical trials have become a hotbed of innovation, often leading the way in adaptive design, technology use, and ...