An exceptionally rare and fatal genetic disease may finally have a solution. Huntington’s Disease affects one in ten to ...

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

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 ...

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

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

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

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

Genetic testing can help doctors diagnose some neurological disorders, such as Huntington’s disease. It can also provide insights into a person’s risk of future health conditions. Genetic testing ...

Rare genetic disorders are typically caused by a variant in a single gene. It is estimated that there are over 7000 such conditions, meaning that although individually rare, they are collectively ...

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