PHEMI-Phenylbutyrate in Patients With Lactic Acidosis: A Pilot, Single Arm, Phase I/II, Open-Label Trial.

Purpose: The 6 months pilot, single arm, phase I/II, open-label clinical trial PHEMI investigated the safety and efficacy of daily administration of phenylbutyrate in reducing lactic acidosis by at least 20% in 3 children (ages 7-10 yrs) with pyruvate dehydrogenase deficiency and 6 adults with mitochondrial myopathy encephalopathy lactic acidosis and stroke-like episodes. As a side study, we investigated the response to phenylbutyrate treatment in skin fibroblasts and cybrids derived from PHEMI patients with the aim of unraveling a possible in vivo-in vitro correlation.

Methods: Safety was assessed through the collection of vital signs, clinical evaluations, blood samples, and reported adverse events.

Micro-encapsulated and colonic-release sodium butyrate modulates gut microbiota and improves abdominal pain in patients with symptomatic uncomplicated diverticular disease.

The role of gut microbiota (GM) in the pathogenesis of Symptomatic Uncomplicated Diverticular Disease (SUDD) remains controversial. Here, we assessed the efficacy of a butyrate formulation in modulating GM and abdominal pain in patients with SUDD. A retrospective study was conducted in patients with SUDD who were treated with a delayed- and colonic-release formulation of butyrate (two capsules bid, for a total dose of 400 mg butyrate) for 3 months.

A prospective multicenter study on prognostic factors and quality of care in Severe Acquired Brain Injury rehabilitation units: a project from the Tiresia network.

Background: Severe Acquired Brain Injury (sABI) presents significant challenges in clinical management and rehabilitation due to its diverse and complex nature. The primary objective of the Tiresia project is to identify medium and medium-to-long-term prognostic factors for patients with sABI and to develop outcome indicators to evaluate the quality of care in rehabilitation units.

Methods: This paper outlines the protocol for a prospective observational multicenter study conducted within the Tiresia Network.

Multiple office blood pressure monitoring for the diagnosis of hypertension in children.

Unlabelled: The measurement (M) of blood pressure (BP) in children may be challenging, and the ideal approach, free of critical issues (such as the interference due to the white coat effect or to the high variability), has yet to be identified. Herein, we compare BP as measured with multiple office BP monitoring (mOBPM) with the standard approach as suggested by guidelines. A cohort of healthy children underwent two mOBPMs 1 year apart.

miRNA-503 inhibition exerts anticancer effects and reduces tumor growth in mesothelioma.

Background: Malignant mesothelioma (MM) is a rare and aggressive form of cancer that affects the mesothelial surfaces, associated with exposure to asbestos fibres. To date, no cure is available for MM and therapeutically approved treatments are based on the use of platinum compounds often used in combination with other drugs. We have previously analysed the efficacy of a cisplatin/piroxicam (CDDP/P) combined treatment showing that this treatment was able to reduce in vivo tumor growth.