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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
Vaccine inequity, inconsistent public health measures and new variants such as Omicron are prolonging the COVID-19 pandemic, but controlling the virus remains possible.
The COVID-19 pandemic continued to rage in 2021, but this was increasingly an unequal pandemic, with rich countries receiving millions of doses of vaccine and ending lockdowns and travel bans. It was also a year when policymakers started to prepare for the next inevitable pandemic.
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.
The FREEDOM studies evaluated exenatide delivered via an implanted minipump in patients with type 2 diabetes; the final study evaluated cardiovascular outcomes but offers up more questions than answers.