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

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

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.

Single-cell proteomics with RNA sequencing uncover hidden gene expression dynamics in stem cell differentiation.

In 1957, just four years after Francis Crick and other scientists solved the riddle of DNA’s structure—the now famous double ...

DAWNZERA is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks ...

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of "genetic surgery" is performed on DNA, for ...

Researchers at San Diego State University and Michigan State University are shedding new light on how viruses meticulously ...

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

As aging progresses, the quality of DNA and proteins inside cells declines, which is known to be the cause of various ...

Waltham, Massachusetts–based Skyhawk Therapeutics has been collecting collaborations with larger companies in spades since ...