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

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

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

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

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

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

The mRNA platform enabled scientists to begin work on a vaccine within days of the publication of COVID-19’s genetic sequence. Clinical trials for an mRNA vaccine began weeks la ...

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 ...

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

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 ...

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

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