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.

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

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

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

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

In the last 86 years, the lethal outcome has not changed. ALS is a cruel, paralyzing and 100% fatal disease. But today, the ALS community has hope. A coalition of ALS patients and family members has ...

If you think you are seeing more ticks, you are not imagining things. With the growth of the deer population come more ticks, which can spread maladies like Lyme disease and Rocky Mountain spotted ...

PUEBLO, Colo. (KKTV) - Izzabella “Izzy” Martin is just 19 years old, but four years ago, she found out she had this rare ...

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