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

September is PKD awareness month, and it’s an opportunity to spread the word about polycystic kidney disease. PKD affects ...

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

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

Four American families are desperate to save their children from a devastating genetic disease. The cure exists — but it’s up ...

A new gene therapy treatment developed by uniQuire has shown promise in reducing the progression of Huntington’s Disease by ...

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 ...

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

Inherited heart conditions place millions of children at risk from birth. Up to 80% of premature cardiovascular deaths are ...

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

Huntington’s disease has been successfully treated for the first time using a gene therapy, which may be available in the US ...

An experimental gene therapy was found to slow the progression of Huntington's disease, marking a major step toward a ...