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.

Ultra-rare disorders are those affecting fewer than one person in 50,000. Sometimes, indeed, they are unique. And unique ...

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

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

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

While genetic testing may be diagnostic, ordering the tests requires physicians to consider the possibility of the illness first. Treatments Curative treatments are rare but available for diseases ...

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

Jason Selvey died six years before his son, Daniel, due to the rare genetic illness Alagille Syndrome ...

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

The genetic modification therapy market presents key opportunities in developing specialized treatments for rare, inherited, ...