A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

When two defective gene variants combine, normal protein function can sometimes be restored.

Pam Kowalcyzk and her daughter have PKU, a rare disorder that prevents the body from processing protein ...

Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...

Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...

Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...

A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...

Melinda Collier told Newsweek that "you can't and shouldn't chalk up someone, let alone a child, as a lost cause." ...

Hani Duglof says even though he applied for asylum 12 years ago, and his case is still pending, federal agents told him he ...

Doctors at Gandhi Hospital successfully performed a complex heart surgery on a 25-year-old woman with Bardet-Biedl syndrome.

The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates ...

A rare form of diabetes affecting newborn babies and caused by a genetic disorder has been discovered by scientists in the UK ...