This phase 2 trial showed that a single subcutaneous dose of L9LS at the highest dose tested (300 mg) provided protective efficacy of 69.9% against P. falciparum infection and 77.4% against ...

Patients with relapsed or refractory hematologic cancers have a poor prognosis. Chimeric antigen receptor (CAR) T-cell therapy as a bridge to allogeneic hematopoietic stem-cell transplantation ...

A total of 52 patients with transfusion-dependent β-thalassemia received exa-cel and were included in this prespecified interim analysis; the median follow-up was 20.4 months (range, 2.1 to 48.1 ...

Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats ...

Most moderate-to-late–preterm infants need nutritional support after birth pending a sufficient supply and intake of mother’s breast milk; however, evidence for the best strategy for nutrition ...

This Journal feature begins with a case vignette highlighting a common clinical problem. Evidence supporting various strategies is then presented, followed by a review of formal guidelines, when ...

In the year after the elimination of a waiver requirement to prescribe buprenorphine, the number of prescribers increased above the anticipated value, but the number of persons who received the ...

Dr. Jamie Aron (Emergency Medicine): A 27-year-old man was evaluated at this hospital because of leg weakness. The patient had been in his usual state of health until 3 days before the current ...

A 57-year-old woman presented with a 3-day history of shortness of breath and dizziness. Laboratory studies showed severe anemia with hemolysis, along with cellular abnormalities in peripheral blood.

The autologous cellular therapy exagamglogene autotemcel is generated by editing an erythroid-specific enhancer of BCL11A. Could another site be edited unintentionally? This study gauged the ...

Disclosure forms provided by the authors are available with the full text of this editorial at NEJM.org. From the Global Health Division, Bill and Melinda Gates Foundation (J.M.M.), and the Fred ...

Recent approvals of exa-cel for treatment of sickle cell disease and transfusion-dependent β-thalassemia mark the dawn of the era of gene editing in medicine. But ensuring access will be challenging.