I was born with a rare genetic disorder, one of about 30 patients worldwide. The hallmark symptoms include a shorter stature, ...

Freya 4, has been diagnosed with a rare metabolic disorder called hereditary fructose intolerance (HFI) ...

After being told her daughter wouldn’t survive, April Perri is now helping her 16-year-old — who has an ultra-rare genetic ...

A new study suggests that certain genetic diseases may be treatable with carefully matched vitamins, including a deadly ...

Doctors at Artemis Hospitals have successfully treated India's first MYSM1-related bone marrow failure syndrome case using a ...

The Food and Drug Administration accepted Opus Genetics's investigational gene therapy into its Rare Disease Evidence Principles program, the company said Monday. The clinical-stage biopharmaceutical ...

The active ingredient in Viagra, known as a treatment for erectile dysfunction, has emerged as a potential candidate for ...

Small changes in genes can make us unique, and they can also have biological effects. Scientists have identified a genetic mutation in a gene called SMIM1 that encodes for a blood group called Vel, ...

Regeneron will make its newly approved gene therapy available for free in the U.S. The company hasn't yet decided how much it will charge in other countries.

Kelly Anne is a deputy editor at Forbes Advisor overseeing the development of various initiatives, including newsletters, social media, and new content verticals. Previously, she worked as a senior ...