Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.

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

This penguin’s features are very special and rare. @bbcearth explains, "This Gentoo penguin's stark white [color] is the ...

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

On Thursday, January 8, Quick and CNBC launched their new initiative, CNBC Cares, to help raise awareness for rare diseases ...

Lurie Children’s received an $11 million gift to expand genetics and rare disease care. The new division aims to shorten diagnostic wait times and increase clinical trials. Officials say the ...

Meet the luckiest people on Earth — individuals carrying a rare genetic mutation that prevents wrinkles from forming even as they age well into their 80s. These genetic lottery winners have provided ...

A local two-month-old baby has a road full of unknowns ahead of her after being diagnosed with a rare genetic condition. Kira ...

Tiffany Fransen, diagnosed with Friedreich's ataxia, faces challenges from this rare disease. The genetic disorder affects ...