Biotech stock KalVista has soared recently, as the firm looks to fundamentally change the treatment landscape for HAE ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

The chronic, progressive disease affects the liver and inflames the bile ducts, causing liver damage. Patients eventually ...

An infant in Philadelphia has been successfully treated with a customized Crispr gene-editing therapy for a rare and deadly disease, doctors reported Thursday—a breakthrough in the futuristic ...

A rare disease is usually defined as a disease or condition that affects fewer than 200,000 people in the United States. According to most scientists and clinicians, there are around 7,000 ...

Doctors have warned that cases of an extremely rare cancer are shooting up with symptoms often mistaken for a common illness ...

A rare and deadly disease has killed dozens of people in the past few months, and authorities in Far North Queensland are struggling to work out what's behind the unprecedented outbreak.

A 14-year-old South Carolina boy has died of a rare bacterial infection within days of showing signs of symptoms. William ...

A life-saving meningococcal B vaccination program will be free for all Tasmanian infants under a re-elected Tasmanian Liberal ...

A DAD-OF-TWO has claimed he nearly died after contracting a rare disease from a “dirty” 4-star hotel. Richard Moore, 55, ...