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 ...

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

“Time is neurons” for 10-year-old Langford girl Charleigh Pollock. Living with the rare neurological disorder Batten disease, ...

A study suggests common psychiatric meds for anxiety, depression or sleep may slightly raise ALS risk later. But experts say ...

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 ...

A rare genetic disease has changed Connor Peebles' life - but what's worse is that his baby son is also affected. Both Connor ...

Spontaneous intracranial hypotension is a rare and often overlooked condition that could have cost one patient her life.And ...