In June, the monoclonal antibody garadacimab (Andembry) was approved for prevention of HAE attacks at a dose of once monthly ...

Sarepta is facing a number of issues currently, including platform-level safety concerns, management credibility issues, etc.

Studying the process of brain formation illuminates just how much of development is a series of tiny miracles. Only a few ...

The approval makes donidalorsen the only treatment in the US for hereditary angioedema that is an RNA-targeted prophylactic.

On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Dawnzera ...

The quest for a vaccine against HIV has been going on for about 40 years — almost as long as the virus that causes AIDS has ...

In the past decade, there has been significant interest in studying the expression of our genetic code down to the level of ...

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not ...

By late 2021, mRNA vaccines had saved an estimated 7.7m lives globally, including most of the 3m Americans whom the Commonwealth Fund, an American health-care charity, estimates were saved by vaccines ...

Dawnzera, a prekallikrein-directed antisense oligonucleotide, is supplied as a 80mg/0.8mL solution in a single-dose autoinjector.

Since 2008, the FDA has approved 11 treatments for a rare genetic condition called hereditary angioedema (HAE), three of ...

Developed by Ionis Pharmaceuticals, Dawnzera is the first RNA-targeted medicine approved for HAE, designed to target plasma ...