Optimisation of a novel series of ENaC inhibitors, leading to the selection of the long-acting inhaled clinical candidate ETD001, a potential new treatment for cystic fibrosis.

Cystic Fibrosis (CF) results from the loss of function of the cystic fibrosis transmembrane conductance regulator (CFTR), an ion channel of key importance in the airway epithelia. CFTR helps control optimal hydration of the airways, a crucial requirement for healthy lungs. CFTR modulators have recently been approved as an effective treatment option for many genetic variants of CF.

Exploratory evaluation of pharmacodynamics, pharmacokinetics and safety of rivaroxaban in children and adolescents: an EINSTEIN-Jr phase I study.

Background: The EINSTEIN-Jr program will evaluate rivaroxaban for the treatment of venous thromboembolism (VTE) in children, targeting exposures similar to the 20 mg once-daily dose for adults.

Methods: This was a multinational, single-dose, open-label, phase I study to describe the pharmacodynamics (PD), pharmacokinetics (PK) and safety of a single bodyweight-adjusted rivaroxaban dose in children aged 0.5-18 years.

Increased neonatal platelet deposition on subendothelium under flow conditions: the role of plasma von Willebrand factor.

In vitro platelet function of umbilical cord blood and neonatal peripheral vein blood from full-term newborns was compared with that of adults. Citrated whole blood was subjected to shear stress (1300 s(-1)) on subendothelial extracellular matrix (ECM)-coated wells in a cone and plate(let) analyzer. Adhered platelets on the ECM were quantitated by image analyzer.

Aurioscope earpieces--a potential vector of infection?

Objectives: to determine whether non disposable aurioscope earpieces used in community paediatric clinics harbored pathogenic micro-organisms, and to explore primary pediatrician perception of the possibility of cross infection from contaminated aurioscope earpieces and of how their aurioscope earpieced are cleaned.

Design: randomized survey.

Setting: four community pediatric clinics.

Plain radiography for diagnosis of gastrointestinal perforation in immune-compromised children--is it enough?

Gastrointestinal perforation is a surgical emergency in the pediatric patient and any delay in diagnosis might be hazardous. In immunocompromised children, the clinical signs of perforation may be blunted. We describe a child with acute lymphoblastic leukemia (ALL) and a perforated appendix and ileum in whom computerized tomography (CT) revealed extraluminal air that was not initially identified on plain abdominal film.