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CERT1 mutations perturb human development by disrupting sphingolipid homeostasis
(2023-05-15)
Neural differentiation, synaptic transmission, and action potential propagation depend on membrane sphingolipids, whose metabolism is tightly regulated. Mutations in the ceramide transporter CERT (CERT1), which is involved ...
Genotype-phenotype relationships of truncating mutations, p.E297G and p.D482G in bile salt export pump deficiency
(2023-02)
Background & Aims
Bile salt export pump (BSEP) deficiency frequently necessitates liver transplantation in childhood. In contrast to two predicted protein truncating mutations (PPTMs), homozygous p.D482G or p.E297G mutations ...
A pooled subgroup analysis of glucarpidase treatment in 86 pediatric, adolescent, and young adult patients receiving high-dose methotrexate therapy in open-label trials
(2023-09)
Background
Delayed methotrexate elimination can occur in patients undergoing high-dose methotrexate cancer treatment. Effectiveness of glucarpidase for rapidly reducing methotrexate concentrations was shown in compassionate-use ...
Adjuvant nab-Paclitaxel + Gemcitabine in Resected Pancreatic Ductal Adenocarcinoma: Results From a Randomized, Open-Label, Phase III Trial
(2023-04-10)
PURPOSE
This randomized, open-label trial compared the efficacy and safety of adjuvant nab-paclitaxel + gemcitabine with those of gemcitabine for resected pancreatic ductal adenocarcinoma (ClinicalTrials.gov identifier: ...
Reliability assessment of ultrasound muscle echogenicity in patients with rheumatic diseases: Results of a multicenter international web-based study
(2023-01-17)
Objectives: To investigate the inter/intra-reliability of ultrasound (US) muscle echogenicity in patients with rheumatic diseases.
Methods: Forty-two rheumatologists and 2 radiologists from 13 countries were asked to ...
2023 WSES guidelines for the prevention, detection, and management of iatrogenic urinary tract injuries (IUTIs) during emergency digestive surgery
(2023-09-09)
Iatrogenic urinary tract injury (IUTI) is a severe complication of emergency digestive surgery. It can lead to increased postoperative morbidity and mortality and have a long-term impact on the quality of life. The reported ...
BT595, a 10% Human Normal Immunoglobulin, for Replacement Therapy of Primary Immunodeficiency Disease: Results of a Subcohort Analysis in Children
(2023-04)
Purpose
To assess the efficacy, pharmacokinetics, and safety of a new, highly purified 10% IVIg (BT595, Yimmugo®) administered in children with PID.
Methods
This was an open-label, prospective, uncontrolled, multicenter ...
Fully resorbable poly-4-hydroxybutyrate (P4HB) mesh for soft tissue repair and reconstruction: A scoping review
(2023-04-12)
Background: Poly-4-hydroxybutyrate (P4HB) is a fully resorbable, biologically-produced polymer with a strength and flexibility comparable to permanent synthetic polymers. The objective was to identify/summarize all ...
Antimicrobial Defined Daily Dose in Neonatal Population: Validation in the Clinical Practice
(2023-03-17)
Background: Currently, there is no validated method for estimating antimicrobial consumption in the neonatal population, as it exists for adults using Defined Daily Doses (DDD). In neonatology, although there are different ...
A Phase 1/2A trial of idroxioleic acid: first-in-class sphingolipid regulator and glioma cell autophagy inducer with antitumor activity in refractory glioma
(2023-09-21)
Background
The first-in-class brain-penetrating synthetic hydroxylated lipid idroxioleic acid (2-OHOA; sodium 2-hydroxyoleate), activates sphingomyelin synthase expression and regulates membrane-lipid composition and ...