Duchenne muscular dystrophy (DMD) is an X-linked genetic disease of skeletal and cardiac muscle that leads to loss of ambulation and premature death due to progressive myopathy and cardiomyopathy. Deramiocel, a heart-derived cellular therapy consisting of human allogeneic cardiosphere-derived cells, improved cardiac and skeletal muscle function in phase 1-2 studies of DMD. Our aim was to assess the efficacy and safety of deramiocel in advanced DMD and support the findings of HOPE-2.

Pulmonary arterial hypertension (PAH) is a rare, progressive disease characterised by elevated pulmonary vascular resistance that can lead to right ventricular failure and premature death. Ralinepag is an oral, once-daily, selective prostacyclin IP receptor agonist developed to treat PAH. We aimed to evaluate the efficacy and safety of ralinepag in patients with PAH.

WHO recommends 5 days of intravenous antibiotics for children hospitalised with severe community-acquired pneumonia (CAP). We aimed to investigate the safety of a step-down to different oral antibiotics and the shortest effective duration.

Mortality among infants with severe HIV-associated pneumonia remains high. This trial (EMPIRICAL) tested whether empirical valganciclovir treatment for cytomegalovirus improves survival in infants in Africa.

Venetoclax-rituximab (VR) following covalent Bruton tyrosine kinase (BTK) inhibitor therapy is the fixed-duration standard of care for patients with chronic lymphocytic leukaemia (including small lymphocytic lymphoma). Pirtobrutinib, a non-covalent BTK inhibitor, is approved for use after treatment with a covalent BTK inhibitor as a continuous therapy option. We aimed to evaluate the addition of pirtobrutinib to VR as a fixed-duration regimen in patients with relapsed or refractory chronic lymphocytic leukaemia.

Currently children's needs, perspectives, and rights are not adequately included in public policies, with negative consequences for the health and wellbeing of children and future generations. The 2020 WHO-UNICEF-Lancet Commission reviewed threats to children's health and concluded that children's needs and voices should be centred in all policies for a sustainable future. Since 2021, Children in All Policies 2030, a global collaboration of policy makers, scientists, and advocates, has implemented the Commission's recommendations by fostering new approaches to participatory, intersectoral policy making across diverse countries. Efforts to implement the Commission's recommendations encountered challenges including flawed assumptions in prevailing policy-making models, failure to fulfil children's right to participate, and an ongoing scarcity of intersectoral policy integration. Eight lessons on what works for improving policy making and implementation emerged: use creative means to involve children, patiently assemble coalitions, prepare to seize political opportunities, harmonise global data to bridge UN partnerships, create national political and technical platforms, use media to change cultural perceptions, use strategic framing to overcome sectoral barriers, and embrace joint learning. Harnessing people's consideration and concern for children and future generations and engaging children's voices represents a powerful political opportunity to reach current development goals and ensure a healthier, more sustainable future.

Dislocation is the most common early surgical complication in patients with hip fractures treated with total hip replacement (THR). Dual mobility total hip replacement (DM-THR) was developed to increase joint stability, but no randomised trial has investigated its efficacy and safety. We aimed to compare the risk of dislocation in patients receiving DM-THR versus conventional THR.

Effective treatments are available for obesity and for hypertension and hypercholesterolaemia, which mediate the cardiovascular and renal effects of obesity. Our aim was to compare blood pressure, cholesterol, and the use of antihypertensive and lipid-lowering medicines in people with obesity and normal weight and assess whether the BMI-associated excess risk has diminished.

Human papillomavirus (HPV) vaccination is a key component of global cervical cancer elimination strategies. While substantial declines in cervical cancer incidence have been observed in several countries, evidence of its effect on mortality is scarce. England introduced a national HPV vaccination programme in 2008 for girls aged 12-13 years (achieving 80-90% coverage before the COVID-19 pandemic), with a catch-up campaign in 2008-10 for girls aged 14-18 years. We aimed to investigate trends in cervical cancer mortality and to estimate the reduction in cervical cancer deaths in young women in England following the introduction of HPV vaccination.

There is conflicting evidence regarding the merits of patellar resurfacing during total knee replacement (TKR), as previous randomised controlled trials (RCTs) have been under-powered and with follow-up of ten years or less.

Previously considered rare, penicillin-susceptible Staphylococcus aureus (PSSA) bacteraemia has re-emerged worldwide. Although benzylpenicillin might offer advantages in terms of pharmacokinetic and adverse effect profiles, anti-staphylococcal penicillins are recommended for serious infections because of concern over undetected penicillin resistance. We aimed to compare benzylpenicillin with anti-staphylococcal penicillins (cloxacillin or flucloxacillin) for the treatment of PSSA bacteraemia in adults.

A growing number of patients with multiple myeloma are anti-CD38 antibody-exposed and lenalidomide-exposed at first relapse, subsequently limiting their treatment options. Mezigdomide, a potent cereblon E3 ligase modulator, induces maximal, rapid Ikaros and Aiolos degradation, resulting in enhanced myeloma cell cytotoxicity and immune stimulation versus immunomodulatory drugs. The SUCCESSOR-2 trial evaluates the efficacy and safety of mezigdomide in combination with carfilzomib and dexamethasone versus carfilzomib plus dexamethasone.

Elecoglipron (AZD5004) is an oral small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist administered once daily without food or fluid restriction, in development for weight management in people living with obesity or overweight and type 2 diabetes. We assessed the efficacy, safety, and tolerability of elecoglipron versus placebo in participants with obesity or overweight and at least one weight-related condition without diabetes.

Approximately 40% of patients with high-risk diffuse large B-cell lymphoma (DLBCL) are not cured with first-line R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone or prednisolone). We aimed to investigate the addition of tafasitamab (an Fc-enhanced anti-CD19 monoclonal antibody) and lenalidomide to R-CHOP (tafa-len-R-CHOP) in patients with high-risk aggressive B-cell lymphomas.

Tau PET imaging has emerged as a critical biomarker for Alzheimer's disease, informing diagnosis, staging, and therapeutic selection. We investigated whether PET tracer selection alters tau detection.

The ORATORIO trial showed that ocrelizumab reduced the risk of disability progression versus placebo in patients with primary progressive multiple sclerosis (PPMS). We aimed to elucidate the effect of ocrelizumab in older and more disabled patients with PPMS, particularly regarding hand function preservation.