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

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

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 ...

Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.

Genome editing-based therapies typically aim to treat disease by correcting underlying genetic mutations in patient's cells.

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 ...

The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism symptoms.

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

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 ...

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 ...