Past the red carpets and the flashing lights of the paparazzi, some celebrities live in a reality more complicated than what ...

It is probably safe to say that progressive supranuclear palsy is not a household name. Unlike Parkinson’s or Alzheimer’s, conditions that have become part of the common vocabulary, progressive ...

Queen’s University has launched a new online resource to empower unpaid carers supporting their loved ones with a rare ...

Ipsen’s failure comes shortly after Regeneron’s FOP study met its endpoints, sparking an FDA approval submission.

Researchers report promising results for nebokitug, a monoclonal antibody tested for primary sclerosing cholangitis. In a Phase 2 trial, the drug proved safe and showed signs of reducing liver ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

Rare blood cancers, once less studied, are now gaining fresh scientific momentum that is driving breakthroughs in disease understanding and treatment ...

VHL, a genetic cancer condition, recently was featured on "Grey's Anatomy." Feb. 10, 2011 — -- In a multi-episode arc on "Grey's Anatomy," actor Scott Foley plays a von Hippel-Lindau (VHL) ...

Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...