Researchers at VIB and Antwerp University have identified a major genetic risk factor for a rare form of frontotemporal ...

The Israeli Basket of Health Services now includes preimplantation genetic testing and in vitro fertilization (PGT-IVF) for individuals carrying three specific genetic markers.

Sildenafil – an active ingredient also marketed under the name of Viagra – improves symptoms in patients with Leigh syndrome. This has now been reported in the Cell* journal by researchers at Charité ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

Nick Jonas' neighbour Maya Kibbel recently died of Wilson's disease at the age of 30. The disease is a rare genetic disorder where copper gets accumulated in the body, affecting different organs.

Genetic diseases are notoriously challenging to treat, especially when each condition requires a tailored approach. With over 10,000 known genetic disorders, developing individual therapies for each ...

Sildenafil—an active ingredient also marketed under the name of Viagra—improves symptoms in patients with Leigh syndrome. This has now been reported in the journal Cell by researchers at ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

Mayo Clinic researchers have identified a rare mutation in the MET gene that can directly cause metabolic dysfunction-associated steatotic liver disease. The mutation disrupts the liver’s ability to ...

Maya was widely known among fans of the Jonas family and was often referred to as Nick Jonas’ “sister-like friend.” Her passing has sparked conversations online about Wilson’s disease, a rare but ...

Donor candidates have to be aged 18 to 60, blood type 0, in good general health, and be willing to undergo screening.

Hereditary ATTR-CM can run in families. Learn who may be at risk, when genetic testing is recommended, the benefits of screening, and how to talk with relatives.