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 ...

The centre, the first of its kind at a government hospital in Maharashtra, was established under the state’s Maternal and ...

Schizophrenia (SZ) and Autism Spectrum Disorders (ASD) are two distinct heterogenous disorders that despite having a ...

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

Kangaroo Island koalas appear safe, but genetic studies reveal hidden risks. Their survival depends on action now.

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 ...

A Columbus family is on a mission to find a cure for a rare genetic disorder and Saturday morning was their third annual Hope With Lo 5k. “We believe that hope isn’t ...

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 ...