Dossier
The New England Journal of Medicine
Coverage of The New England Journal of Medicine in the Nexus archive.
Daraxonrasibtopic1Dana-Farber Cancer Instituteorganization1Brian Wolpinperson1Hale Family Center for Pancreatic Cancer Researchorganization1Food and Drug Administrationorganization1Otarmeni (lunsotogene parvec-cwha)topic1OTOF genetopic1FDA Commissioner Marty Makaryperson1National Priority Voucher (CNPV) pilot programorganization1Otoferlintopic1Cochleatopic1
- New pancreatic cancer pill could reshape treatment as early trial results stun researchers
A new pancreatic cancer pill called Daraxonrasib has shown promise in early testing, with about 30% of patients seeing a positive response and 90% having their cancer either shrink or stop getting worse. The pill is designed to block multiple active cancer signals that help tumor cells grow. The clinical trial was led by the Dana-Farber Cancer Institute.
- First-ever gene therapy for hearing loss gets FDA approval: 'Groundbreaking'
The FDA approved Otarmeni, the first dual adeno-associated virus (AAV) gene therapy for genetic hearing loss linked to the OTOF gene. The treatment, administered surgically to the inner ear, improved hearing in some patients with OTOF-related deafness, marking the first disease-modifying therapy for this condition.