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

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

In May 2025, researchers announced that K.J. Muldoon, a baby boy born without the ability to process dietary protein properly ...

When you purchase through links on our site, we may earn an affiliate commission. Here’s how it works. A woman's heart problem turned out to be related to a rare genetic disorder.(Image credit ...

Many rare disease patients do not receive a formal genetic diagnosis, but a European data-sharing project aimed to give them a second chance. Arne Cavents was four years old when he was misdiagnosed.

Clinical Trials for Rare Diseases: Challenges, Innovations, and Hope for the Future Clinical trials are the foundation of modern medicine, guiding the development of safe and effective treatments for ...

The resolution reinforces the understanding that addressing rare diseases is essential to achieving universal health coverage and to building equitable, people-centered health systems,” Virginie ...

The company is on track to complete IND-enabling studies and submit an IND to FDA in 2026. For more information about Cure Rare Disease and its research pipeline, please visit www.cureraredisease.org.

Of the more than 10,000 known rare diseases, less than 10% have an FDA-approved treatment. That is why, of the more than 10,000 known rare diseases, less than 10% have an FDA-approved treatment.

Abeona Therapeutics Inc. announced the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $155 million, following FDA approval of its treatment ZEVASKYN™. This sale is expected ...

Let’s explore how these remarkable AI tools are revolutionizing rare disease detection and why they might represent the most significant advancement in diagnostic medicine since the invention of ...