All articles tagged with #nejm
A NEJM case report documents a young Hurler syndrome patient who received adeno-associated virus–delivered gene therapy and later developed a walnut-sized brain tumor believed to be caused by an unintended gene mutation; the tumor was removed, cognitive development progressed, highlighting rare but serious risks associated with gene therapies.
A five‑day course of ensitrelvir (Xocova) significantly reduced the risk of developing COVID-19 after household exposure in a double‑blind NEJM phase 3 trial of about 2,000 participants: 9% in the placebo group developed symptoms within 72 hours versus 2.9% in the drug group; by day 10, 14% vs 21.5% tested positive. Side effects were similar between groups. The drug is already approved in Japan for treatment and is being considered for post‑exposure prevention by US/EU regulators; pregnancy safety was not studied.
A New England Journal of Medicine analysis of Canadian and UK trials (about 4,200 participants) found Paxlovid does not reduce hospitalizations or death in a largely vaccinated population, though users recovered about three days faster; the results could influence how public funding and prescriptions are targeted for antivirals.
Ancient DNA analysis of two closely related Ice Age skeletons from Romito Cave in Italy identifies a mother–daughter pair, with the younger diagnosed with acromesomelic dysplasia, Maroteaux type (AMDM) caused by NPR2 mutations—the earliest known AMDM case. The study highlights how a family carried a gene variant affecting bone growth and shows care within the group; findings were published in The New England Journal of Medicine.
FDA officials say a single pivotal trial will be the default standard for drug approvals, a policy shift outlined in an NEJM perspective by Marty Makary and Vinay Prasad.
The article summarizes recent developments in scientific publishing, including NEJM launching a new public health report rival, a former NIH official's paper receiving an expression of concern, and a study revealing that 1 in 5 chemists have intentionally added errors during peer review, highlighting ongoing issues of misconduct and integrity in research.
The NEJM and CIDRAP are launching a new, free publication of public health alerts as an alternative to the CDC's MMWR, aiming to improve timely dissemination of outbreak data amid declining trust and operational challenges faced by the CDC's traditional publication.
The National Institutes of Health (NIH) has implemented a new data sharing policy to increase transparency in biomedical research, a move praised in a recent New England Journal of Medicine (NEJM) op-ed. This policy shift marks a significant reversal from NEJM's previous stance, which had controversially criticized data sharing by coining the term "research parasites." The change reflects the growing Open Science movement, advocating for public access to data from government-funded research to accelerate scientific progress. The NEJM now supports the idea of a government repository for scientific data to ensure compliance with sharing policies and facilitate broader research collaboration.