By focusing on twins and patients with lupus and Parkinson’s disease, a Mexican consortium is generating critical data to ...

More than 1,000 genes may serve as possible treatment targets for individuals with kidney disease, according to a new study, published in Science, by researchers at the Perelman School of Medicine ...

Mirum Pharmaceuticals' Ctexli wins FDA approval for CTX, a rare lipid storage disease. The company projects 2025 global sales of $420–$435 million.

MONDAY, Aug. 19, 2024 (HealthDay News) -- All 23 known species of the bacteria that cause Lyme disease have now been genetically mapped, providing an aid to better diagnosis and research. "This ...

A young European royal has died due to a rare genetic disorder at the age of 22. Prince Frederik of Luxembourg passed away from a rare mitochondrial condition affecting two of his POLG genes that ...

The era of genetic medicines has ushered in novel and exciting ways of treating genetic diseases, one of which includes bringing to reality the promise of a one-time treatment by addressing the ...

REYKJAVIK, Iceland, Jan. 23, 2025 /PRNewswire/ -- Scientists at deCODE genetics/Amgen have constructed a complete map of how human DNA is mixed as it is passed down during reproduction. The map ...

A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness.

When a young New York boy was diagnosed with a rare genetic disorder, hope came in an unlikely form — a golden retriever named Yammy. His mother shares the inspiring story with Fox News Digital.

A 20-year-old died while embarking on a family trip as part of his final wish. The Naylor siblings were all born with myotonic dystrophy, a genetic disorder that causes muscles to waste away.

An experimental treatment that stimulates the spinal cord may help people with a paralyzing genetic disorder. NPR's Jon Hamilton reports on a study of three people with a rare inherited condition.