Late on Wednesday, U.S. Food and Drug Administration (FDA) Commissioner Marty Makary and Center for Biologics Evaluation & Research (CBER) Director Vinay Prasad published an article in the New England ...

The cGAS–STING pathway is a component of the innate immune system that functions to detect the presence of cytosolic DNA and, in response, trigger expression of inflammatory genes that can lead to ...

In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therapies ...

This article is the latest in the Health Affairs Forefront featured topic, “Health Policy at a Crossroads,” produced with the support of the Commonwealth Fund and the Robert Wood Johnson Foundation.

A new study suggests a dual mechanism of actions of overactive and cytosolic re-localized BRCA1, the major guardian of genomic stability, in neurons death by aberrant DNA damage response and ...

The US Food and Drug Administration (FDA) is proposing a new “plausible mechanism” pathway that will enable the limited approval of bespoke therapies when randomized trials are impractical. FDA will ...

Personalized genetic interventions like antisense oligonucleotides and genome editing can help children with rare diseases, but it has been difficult for manufacturers to get regulatory approval for ...

Duchenne muscular dystrophy (DMD) is a severe X-linked myopathy characterised by progressive skeletal and cardiac muscle degeneration, loss of ambulation, respiratory failure and premature mortality.