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An antisense oligonucleotide targets mutant transcripts of the ALS gene C9orf72, suppressing the poly(GP) dipeptide in tissues in mice and in the spinal fluid of a single patient harboring the C9orf72 gene mutation
The risk of intensive care unit admission among children with COVID-19 or its sequelae is affected by age, ethnicity and comorbidities. Understanding these modifiers might help identify at-risk children and guide interventions.
In a prespecified interim analysis of a pivotal phase 2 trial, tisagenlecleucel, an autologous CD19-targeting CAR-T cell therapy, produced a high rate of complete responses with a manageable safety profile in adults with relapsed or refractory follicular lymphoma
A self-controlled case series using individual-patient-level data from over 38 million people aged 16 years and over, reveals an increased risk of myocarditis within a week of receiving a first dose of ChAdOx1, BNT162b2 and mRNA-1273 vaccines, which was further increased after a second dose of either mRNA vaccine. SARS-CoV-2 infection was associated with even greater risk of myocarditis, as well as pericarditis and cardiac arrhythmia.
Artificial intelligence algorithms trained using chest X-rays consistently underdiagnose pulmonary abnormalities or diseases in historically under-served patient populations, raising ethical concerns about the clinical use of such algorithms.
A progressive movement disorder in a patient with multiple myeloma treated with anti-BCMA CAR-T cells that might have been related to on-target activity in the brain supports prospective neurologic monitoring after BCMA-targeting therapies.
Biomarker analysis of the phase 3 JAVELIN Bladder 100 trial leads to the development of a multi-parameter model comprising tumor and immune features that might identify patients with advanced urothelial cancer who will benefit from avelumab maintenance therapy.
Single-cell transcriptomic analyses of human fetal and neonatal intestinal cells reveal a subpopulation of enteroendocrine cells that produce insulin during development.
An mRNA vaccine platform to prevent HIV-1 infection generated broadly neutralizing antibodies in non-human primates and protected some animals from infection, raising hope that optimization of this approach might lead to an effective HIV vaccine.
Genomic analyses in individuals with index and familial intracranial aneurysms and experiments in vertebrate models identify pathogenic variants in the PPIL4 gene implicated in cerebral angiogenesis and cerebrovascular integrity, through the Wnt signaling pathway.
A first-in-class immune-modulating IDO/PD-L1-targeting vaccine combined with nivolumab in a phase 1/2 trial achieved very promising clinical activity and long-lasting immune responses in patients with advanced melanoma.
In a phase 1 trial of patients with advanced systemic mastocytosis, avapritinib, a selective KIT inhibitor, was generally well tolerated, elicited durable clinical responses and led to reductions in mast cell disease burden.
In a prespecified interim analysis of a pivotal phase 2 trial, avapritinib, a selective KIT inhibitor, elicited robust clinical and molecular responses, was generally well tolerated and led to improved patient-reported outcomes in patients with advanced systemic mastocytosis.
In patients with type 2 diabetes and at risk for atherosclerotic cardiovascular disease, the FREEDOM Cardiovascular Outcomes study showed that a continuous subcutaneous infusion of exenatide via an osmotic mini-pump was non-inferior compared to placebo in terms of cardiovascular safety outcomes.
In a prospective study, a smartwatch-based alerting system was able to detect pre-symptomatic and asymptomatic SARS-CoV-2 infection in a high percentage of cases.
The multicenter phase 2 TRIUMPH trial shows the utility of ctDNA genotyping as a screening platform to select patients with HER2-amplified metastatic colorectal cancer who benefit from dual-HER2 blockade with trastuzumab and pertuzumab
A clinical review of all pediatric deaths in England from March 2020 to February 2021 attributes 25 deaths to SARS-CoV-2 infection and identifies risk factors to guide interventions.