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 ...

Necrotising fasciitis is a rare but deadly flesh-eating infection. Early treatment is vital to prevent sepsis, amputation, or ...

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

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 ...

Many rare disease patients do not receive a formal genetic diagnosis, but a European data-sharing project aimed to give them a second chance. Arne Cavents was four years old when he was misdiagnosed.

Experts are sounding the alarm over the spread of a rare deadly virus after two Americans tested positive this week.

A dad-of-two claims he nearly died after contracting a rare disease from a 'dirty' 4-star hotel while on a sunshine break in ...

Oxford, UK-based biotech SynaptixBio, which is developing a therapy for a rare, deadly, and currently incurable rare disease (H-ABC), says it has identified the drug it will take forward into clinical ...

KJ Muldoon was diagnosed with a rare, deadly genetic disease after he was born. KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing ...