POLG-related diseases disrupt the function of the mitochondria, or "powerhouses" of the cell — starving them of energy.

Early research shows a one-time gene therapy has potential for addressing both the liver and lung disease that patients with ...

Big cats have long captivated our imaginations with their majestic beauty and powerful presence. However, beyond their ...

A rare genetic mutation causes some lions to develop a breathtaking white coat—but this stunning trait is disappearing fast. Why do white lions exist? What makes them so rare? And why are they ...

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

A research team co-led by scientists at the University of Arizona College of Medicine—Tucson found that an osteoporosis drug might counter a rare genetic mutation underlying a type of heart disease.

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life.

Researchers report practice-based evidence built on clinical findings where an intensive therapeutic approach called ACQUIRE ...

Alltrna’s CEO Michelle Warner said that the approach allows for basket trials that can group patients with different diseases ...

Two more people have been potentially cured of HIV after receiving stem cell transplants to treat blood cancer or bone marrow ...

Pune: The Zika virus strain that circulated in the city between June and September last year had certain concerning mutations ...