Original Article

This phase 1–2 study involving nine children with leukocyte adhesion deficiency type 1 showed significantly better survival with lentiviral gene therapy without hematopoietic stem-cell transplantation than that in historical controls.

Lepodisiran, an extended-duration, small interfering RNA, reduced lipoprotein(a) concentrations in participants with elevated levels.

In six families with familial polycythemia, EPO variants led to the production of a hepatic-like erythropoietin that had an altered glycosylation pattern and was more active than the renal form.

In this trial, the addition of mepolizumab to the treatment of patients with COPD and an elevated eosinophil count led to a lower rate of moderate or severe exacerbations than placebo.

In this interim analysis of a phase 3 trial involving 800 patients with metabolic dysfunction–associated steatohepatitis, once-weekly semaglutide improved liver histologic results at 72 weeks.

Among patients with tumors harboring tyrosine kinase domain mutations who received zongertinib (a selective inhibitor of the HER2 tyrosine kinase), 71% had a response, and median progression-free survival was 12.4 months.

Neoadjuvant PD-1 blockade with dostarlimab led to organ preservation in a high proportion of patients with early-stage mismatch repair–deficient tumors, with no compromise of surgical options.

In this randomized trial conducted over the 2019–2024 influenza seasons, baloxavir resulted in a significantly lower incidence of transmission of influenza virus from patients to household contacts than placebo.

A comparison of clinical outcomes in 39 patients treated with atidarsagene autotemcel, a hematopoietic stem-cell–based gene therapy, with those of untreated patients showed a strong treatment effect over the long term.

In this randomized trial involving patients with noncystic fibrosis bronchiectasis, the rate of pulmonary exacerbations over a 52-week period was lower with brensocatib (10 mg or 25 mg per day) than with placebo.

Both clonal hematopoiesis of indeterminate potential (CHIP) and cancer are more common with age. When they coexist in the same patient, CHIP cells may migrate to the tumor and have an adverse effect on overall survival.

In this trial of the aldosterone synthase inhibitor lorundrostat in participants with uncontrolled hypertension, lorundrostat was associated with greater reductions in blood pressure than placebo.

In this trial, obinutuzumab, a humanized type II anti-CD20 monoclonal antibody, plus standard therapy provided significantly better renal responses than standard therapy alone in patients with lupus nephritis.

In a randomized trial, pulsed field ablation was noninferior to cryoballoon ablation with respect to the incidence of a first recurrence of atrial tachyarrhythmia, as assessed by continuous rhythm monitoring.

In a long-term follow-up study, a single dose of fidanacogene elaparvovec was found to lead to sustained factor IX expression in liver cells and dramatically lowered annualized bleeding rates in 14 patients with hemophilia B.

Treatments for mpox are needed. In this trial in the Democratic Republic of Congo, tecovirimat did not lead to faster resolution of mpox lesions than placebo in patients with clade I MPXV infection.

In a trial involving patients with active cancer and venous thromboembolism, extended anticoagulation with reduced-dose apixaban was noninferior to full-dose apixaban for preventing recurrent thromboembolic events.

In a trial involving patients with ischemic stroke due to medium-vessel occlusion, thrombectomy within 12 hours did not lead to a better functional outcome and lower mortality at 90 days than usual care.

In this trial involving 543 patients with stroke due to occlusion of medium or distal vessels, endovascular treatment within 24 hours after the onset of symptoms was not effective in improving functional outcome at 90 days.

Among older patients undergoing TAVI, dapagliflozin led to a lower incidence of a composite of death from any cause or worsening of heart failure than standard care at 1 year.