Malaria is a major cause of illness worldwide. In a phase 2 trial in Mali, one subcutaneous dose of L9LS, a monoclonal antibody targeting Plasmodium falciparum, reduced the incidence of clinical malaria among children.
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 likelihood of off-target editing.
The Supreme Court has imposed new legal principles that impede the ability of states, Congress, and agencies to use evidence to protect the public. It now has the opportunity to reconsider this approach.
A 30-year-old woman was evaluated because of back pain, leg stiffness, and falling. Tone was increased in the legs. Exaggeration of the normal lumbar lordosis was seen on MRI. What is the diagnosis? Cast your vote. Find the answers in the full text of the case, to be published on May 8.
Of 30 patients with severe sickle cell disease who were treated with gene-edited autologous hematopoietic stem and progenitor cells, 29 were free from vaso-occlusive crises for at least 12 consecutive months.
Of 35 patients with transfusion-dependent β-thalassemia treated with gene-edited autologous hematopoietic stem and progenitor cells, 32 had hemoglobin levels maintained without red-cell transfusions for at least 12 consecutive months.
This podcast episode examines CAR T-cell therapy’s early successes, broader promise, and emerging risks, as the FDA considers reports of occasional secondary cancers.
Original Research
Among patients hospitalized for acute myocardial infarction and at risk for heart failure, empagliflozin did not lead to a significantly lower risk of a first heart-failure hospitalization or death from any cause than placebo.
In a registry-based trial, FFR-guided PCI of nonculprit lesions did not result in a lower risk of a composite of death from any cause, myocardial infarction, or unplanned revascularization than culprit-lesion-only PCI.
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 drug did not.
In a randomized trial involving patients with STEMI and cardiogenic shock, mortality at 6 months was lower with mechanical circulatory support with a microaxial flow pump than with standard care alone.
Of 10 patients with relapsed or refractory hematologic cancers who received sequential CAR T-cell infusion followed by allogeneic stem-cell transplantation without GVHD prophylaxis, 6 were alive and disease-free at last follow-up.
In moderate-to-late–preterm infants, provision of parenteral nutrition, milk supplement, and exposure to taste and smell did not affect body composition at 4 months or time to full enteral feeding.
Improved survival in renal-cell cancer after 1 year of adjuvant therapy was seen with pembrolizumab. At 4 years, 91% of pembrolizumab-treated patients were alive, as compared with 86% of those who received placebo after surgery.
Clinical Practice and Review
A 27-year-old man presented with weight loss, diarrhea, tremor, and proximal muscle weakness. The blood level of potassium was 1.8 mmol per liter. What is the diagnosis?
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.
A 58-year-old woman was transferred to the hospital after 16 months of waxing and waning confusion and aphasia and evolving changes on MRI of the head. What is the diagnosis?
Hearing progressively declines with age, manifesting initially as difficulty understanding speech in background noise and detrimentally affecting social functioning. Strategies and technologies can increase speech-signal clarity.
Despite being absolutely clear that she wanted a quick, painless end of life, a geriatrician’s elderly mother receives aggressive care. How can we ensure that such patient preferences are honored?
The study of retinoblastoma has led to fundamental insights into carcinogenesis, cell-cycle regulation, the genetic basis of cancer, and tumor-suppressor genes, as well as to advances in therapy.
This season finale examines the moral injury that the current U.S. health care system inflicts on physicians and trainees — and how they may be able to offer care that aligns with their values.
