Personalized Gene Editing to Treat an Inborn Error of Metabolism | NEJM
In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder.
Related Articles
Impact of Patient Access to Care on Rates of Febrile Neutropenia Post Inpatient Administration of a Colony-Stimulating Factor
This abstract will be presented at the Oncology Pharmacists Connect (OPC) meeting in Austin, Texas, from June 19 to 20, 2025.
Genomics | bioRxiv
bioRxiv – the preprint server for biology, operated by Cold Spring Harbor Laboratory, a research and educational institution
Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes | NEJM
Zimislecel is an allogeneic stem cell–derived islet-cell therapy. Data on the safety and efficacy of zimislecel in persons with type 1 diabetes are needed. We…
Cagrilintide–Semaglutide in Adults with Overweight or Obesity and Type 2 Diabetes | NEJM
Cagrilintide and semaglutide have each been shown to induce weight loss as monotherapies. Data are needed on the coadministration of cagrilintide and semaglutide (called CagriSema)…
Zongertinib in Previously Treated HER2-Mutant Non–Small-Cell Lung Cancer | NEJM
Innovative oral targeted therapies are warranted for patients with human epidermal growth factor receptor 2 (HER2)–mutant non–small-cell lung cancer (NSCLC). Zongertinib is an oral, irreversible,…
Medical AI and Clinician Surveillance — The Risk of Becoming Quantified Workers | NEJM
Many AI innovations could benefit patients and clinicians. Yet medicine should heed lessons from industries in which AI adoption has resulted in reduced autonomy for…
Coadministered Cagrilintide and Semaglutide in Adults with Overweight or Obesity | NEJM
Semaglutide at a dose of 2.4 mg has established weight-loss and cardiovascular benefits, and cagrilintide at a dose of 2.4 mg has shown promising results…
Cagrilintide–Semaglutide in Adults with Overweight or Obesity and Type 2 Diabetes | NEJM
Cagrilintide and semaglutide have each been shown to induce weight loss as monotherapies. Data are needed on the coadministration of cagrilintide and semaglutide (called CagriSema)…
Impact of a Community-Based Specialty Pharmacist in Identifying Drug Therapy Problems for New Adult Oncology Patients
This abstract will be presented at the Oncology Pharmacists Connect (OPC) meeting in Austin, Texas, from June 19 to 20, 2025.