A new discovery offers hope for Huntington’s disease. This discovery provides hope that a DNA repair process may help slow or ...

A genetic study published on Tuesday offers an important new clue. Researchers found that, between 250,000 and 500,000 years ...

Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

Past research studies have consistently found that there are disparities in the health outcomes of people from different ...

Researchers at Tel Aviv University, led by Prof. Illana Gozes, examined the effects of different mutations in the ADNP protein, which is essential for normal brain development and aging, on the brain ...

A new study has uncovered critical genetic factors that could revolutionize the fight against powdery mildew (PM) in melons, a disease that threatens ...

Biogen and Stoke Therapeutics have agreed to collaborate on Stoke's proposed zorevunersen treatment for the severe genetic epilepsy Dravet syndrome in a deal potentially worth hundreds of millions of ...

The partnership splits the rights to Stoke’s epilepsy antisense oligonucleotide, with up to $385 million in potential payments due to Stoke.

In exchange for $165 million, Biogen now has access to zorevunersen, a potential first-of-its-kind treatment for Dravet ...