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.

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 ...

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

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved ...

Dawnzera is the third medicine cleared by U.S. regulators this year for the rare genetic condition, and the second wholly owned medicine Ionis has brought to market.

Senior author Elias Sayour, M.D., Ph.D., a UF Health pediatric oncologist, said the results reveal a potential new treatment path — an alternative to surgery, radiation, and chemotherapy — with broad ...

A previously unknown virus in farmed Pacific oysters may hold the key to mass die-offs in the multimillion-dollar industry, according to research from the University of British Columbia (UBC) in ...

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 ...

Researchers warn that halting federal contracts for mRNA vaccine research could weaken pandemic preparedness and slow medical advances.

Epigenetic regulation plays a critical role in the reshaping of gene expression programs, ultimately driving cancer ...