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

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

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

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

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

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

A seven-year-old Emirati girl, Mahra Saeed Al Balushi, embodied an inspiring humanitarian story for an Emirati family after ...

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

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