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

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 federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...

GeneDx Holdings WGS announced a sponsored genetic testing program with Zevra Therapeutics to improve diagnosis for patients suspected of having Niemann-Pick Disease Type C (NPC), a rare and ...

Researchers at VIB and Antwerp University have identified a major genetic risk factor for a rare form of frontotemporal dementia. The discovery, published today in Nature Genetics, provides a ...

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 ...

This Ice Age pair of hunter-gatherers were both shorter than average for their population – one of them markedly so – and now we finally know why. A team of researchers from Europe re-examined the ...

Chronic kidney disease (CKD) is a public health challenge that affects more than 800 million people worldwide. 1 CKD can be caused by a variety of disease processes. Many causes are difficult to ...

Chronic gastrointestinal issues and obsessive-compulsive disorder fueled fears around food that eventually developed into avoidant restrictive food intake disorder (ARFID) and anorexia. In 2022, ...

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 ...