Johns Hopkins researchers identified a new target for treating Alzheimer’s disease by focusing on a protein that produces ...

Researchers have identified a brain protein whose absence leads to memory loss and Alzheimer’s-like damage in mice.

Qure N.V. (NASDAQ:QURE) shares are popping on Friday after the company said the FDA will review the data package for AMT‑130, ...

Although the year brought a steady stream of new treatments, tests, and cutting-edge science, experts worry about how ...

Shares of UniQure were higher after the gene therapy company said it had scheduled a Type A meeting with the Food and Drug Administration to discuss a Biologics License Application for the company's ...

The Company expects to provide a regulatory update after the receipt of official meeting minutes.

NEW YORK – Sarepta Therapeutics on Wednesday said it submitted an application to regulators in New Zealand, seeking to begin a first-in-human trial of SRP-1005, the siRNA drug it's developing for ...

Loqus23 Therapeutics Ltd. has nominated LQT-23, a first-in-class allosteric oral small-molecule inhibitor of MSH3/MutSβ, as a development candidate for Huntington’s disease.

An analysis of genetic data from over 900,000 people shows that certain stretches of DNA, made up of short sequences repeated ...

Scientists discovered that aging DNA repeats expand at wildly different speeds—and in some people, the consequences can be devastating. A sweeping genetic study drawing on data from more than 900,000 ...

An analysis of genetic data from over 900,000 people shows that certain stretches of DNA, made up of short sequences repeated ...

Sarepta is also pursuing preclinical programs for Spinocerebellar ataxia type 1 (SCA1) and Spinocerebellar ataxia type 3 (SCA3) and has an exclusive collaboration with Arrowhead Pharmaceuticals to ...