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

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

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

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

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

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

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

Working with agency partner DCX Growth Accelerator, GeneDX takes direct aim at the issue of underdiagnosis of pediatric rare diseases.

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 ...

BENGALURU: Gone are the days when a heart attack or cancer were associated with stress, unhealthy lifestyle and bad eating ...

Large Swedish cohort study finds delusional disorder, acute psychoses, psychosis NOS, and schizoaffective disorder are genetically distinct from SZ, BD, and MD.