Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial.

Introduction: Wolfram syndrome (Spectrum Disorder) is an ultra-rare monogenic form of progressive neurodegeneration and diabetes mellitus. In common with most rare diseases, there are no therapies to slow or stop disease progression. Sodium valproate, an anticonvulsant with neuroprotective properties, is anticipated to mediate its effect via alteration of cell cycle kinetics, increases in p21 expression levels and reduction in apoptosis and increase in Wolframin protein expression.

Prospective validation of ORACLE, a clonal expression biomarker associated with survival of patients with lung adenocarcinoma.

Human tumors are diverse in their natural history and response to treatment, which in part results from genetic and transcriptomic heterogeneity. In clinical practice, single-site needle biopsies are used to sample this diversity, but cancer biomarkers may be confounded by spatiogenomic heterogeneity within individual tumors. Here we investigate clonally expressed genes as a solution to the sampling bias problem by analyzing multiregion whole-exome and RNA sequencing data for 450 tumor regions from 184 patients with lung adenocarcinoma in the TRACERx study.

Findings From the Todurujo na Kadurok (Empowering Youth) HIV Self-Testing and Edutainment Comic Randomized Controlled Trial With Refugee Youth in a Humanitarian Setting in Uganda.

Introduction: Humanitarian settings are underserved by HIV self-testing (HIV-ST).

Methods: We conducted a randomized controlled trial to evaluate the effectiveness of HIV-ST (Arm 1), HIV-ST alongside edutainment comics (Arm 2), and edutainment comics (Arm 3), compared with the standard of care (SOC), in increasing HIV testing with refugee youth aged 16-24 in the Bidi Bidi Refugee Settlement, Uganda. Intervention effects on HIV testing at 3-month follow-up (T2) were assessed using generalized estimating equation models alongside open-ended questions.

Catheter-associated vessel perforation presenting as an occluded lumen: lessons learned and preventive strategy.

A central venous catheter (CVC) was placed in the left internal jugular vein of a woman in her late 20s, who was admitted to our intensive care unit during the management of her abdominal wall sepsis. Two days later, the patient had pain at the insertion site. Check aspiration revealed the presence of aspirate from all the lumens except the distal lumen.