A pioneering gene therapy has successfully slowed the progression of Huntington’s disease by 75% in patients after three ...

Researchers have developed a new lipid for delivering mRNA that is safer and more effective than current technologies. By ...

Harmony Biosciences said on Wednesday its experimental drug for a type of rare genetic disorder failed to meet the main goal ...

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” study, according to drugmaker uniQure, marking a major step toward a potential first genetic ...

Ahn has studied SON since the early 2000s. After the discovery of ZTTK, she co-founded the ZTTK SON-Shine Foundation in 2021 ...

Huntington's Disease is a rare genetic disorder affecting more than 40,000 people in the U.S. It causes physical, mental, and ...

Scientists developed a new nanostructure that triples CRISPR’s ability to enter cells, unlocking even more power to treat ...

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.

The U.S. Food and Drug Administration has declined to approve a higher-dose version of Biogen's drug for a rare genetic ...

KJ Muldoon wasn’t supposed to make it to his first birthday. He was born in August 2024 with carbamoyl phosphate synthetase 1 deficiency, a genetic disorder that afflicts about 1 in 1.3 million ...

Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ZYN002 in Fragile X syndrome (FXS). Fragile X syndrome ...