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

Rare Disease Day 2024 is an important opportunity to raise awareness about rare diseases and the need for research, diagnosis, and treatment options like orphan drugs. Learn the significance of ...

To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research.

A Vancouver Island mother says she’s heartbroken after the B.C. government refused to reinstate funding for the only drug ...

A Sterling family is seeking community support after a "perfectly normal day" changed their lives forever. Jeremy Hunter suffered a severe stroke May 20 that resulted in emergency surgery to remove a ...

With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is ...

Nationally Recognized Texas Children’s Hospital Adds First-In-Class Therapy as a Potential Life-Saving Treatment in ...

In late June, a Grand Canyon National Park concessions employee contracted hantavirus, a rare but often fatal rodent-borne ...

This Grade Two learner at Laerskool Rayton suffers from a rare disease called Fanconi anemia (FA), a rare and inherited ...

June is Alzheimer's and Brain Awareness Month. Colbie Schueneman, 6, is sharing her story with KIF1A, also known as KAND.