The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

A 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment was released ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ...

A baby born with a rare and life-threatening genetic disorder has taken his first steps after receiving a groundbreaking, ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...

It began as a medical mystery that puzzled doctors and parents alike, a baby emitting an unmistakable odour of rotting fish. What sounded like a hygiene issue turned out to be a rare genetic condition ...

U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.

A Mackay mother has described the past few months as the hardest of her life as her newborn daughter remains in hospital ...

A couple with deep ties to the Chicago area is donating $11 million to Lurie Children’s Hospital in hopes of speeding research, treatment and diagnosis for children with rare and genetic disorders.

The National Institutes of Health established the Genomics Research to Elucidate the Genetics of Rare Diseases (GREGoR) Consortium in 2021 with the goal of finding molecular diagnoses for individuals ...