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 ...

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 ...

Rather than give up on rare pediatric disease vouchers due to fears of program abuses that have never come to fruition, I urge lawmakers to review the new data and listen to the more than 130 ...

Rare disease research often involves navigating complex legal landscapes. Incredible, impactful organizations are working hard to find cures, but they can’t do it alone.

Despite increases in rare disease treatments attributable to the Orphan Drug Act, the economic value of rare disease treatments has come under increasing scrutiny. Assessing the value of ...

GlaxoSmithKline decided to shut down its drug development program for rare diseases because it wasn't a "winner." That may have been a good business decision, but it abandoned a miracle.

Dr. Tim Guilliams, cofounder and CEO of Healx, is an advocate for harnessing the power of AI to accelerate treatments for rare diseases. There has been an encouraging trend in our understanding of ...

Rare Disease Day is observed every year on Feb. 28 (or 29 in leap years)—the rarest day of the year, according to National Organization for Rare Disorders.

Rare diseases are serious, complex, usually chronic, often life-limiting and most have no cure. We know of about 7,000 different rare diseases, most with a genetic origin. Many begin in childhood ...