Search results for "Cystic Fibrosi"

Quality Improvement Project to Evaluate Discharge Prescriptions in Children With Cystic Fibrosis

2019

Introduction: Cystic Fibrosis (CF) requires multiple pharmaceutical treatments, elevating the risk of medication errors (ME), which may compromise patient safety. This study aimed to improve the quality of discharge prescriptions (DPs) using indicators following admissions for IV antibiotics in pediatric CF patients. Methods: This project involved a longitudinal observational retrospective descriptive study followed by a longitudinal quasi-experimental prospective phase between January 2013 and December 2016 in CF patients admitted to a London Children's Hospital. The CF pharmacist reviewed DPs. Six rights of medication administration were defined (6R): dose, drug, frequency, duration of tr…

Rescuing CFTR Protein Function: 1,3,4-oxadiazoles versus 1,2,4-oxadiazoles as readthrough inducing drugs

In Cystic fibrosis (CF) disease nonsense mutations in the CFTR gene cause the absence of the CFTR protein expression and a more severe form of the disease. About 10% of patient affected by CF show a nonsense mutation. A potential treatment of this alteration is to promote translational readthrough of premature termination codons (PTCs) by translational readthrough inducing drugs such as Ataluren (1). We reported a rationale for Ataluren promoted readthrough of PTCs by computational approach and GFP-reporter cell-based assay (2) and the observed enhancement of readthrough activity by some Ataluren derivatives (3, 4). In this context we aimed to compare the 1,2,4-oxadiazole core of Ataluren w…

OXADIAZOLE DERIVATIVES FOR THE TREATMENT OF GENETIC DISEASES DUE TO NONSENSE MUTATIONS

2018

Are disclosed oxadiazole derivatives, their use as medicaments and in particular for the treatment of diseases associated with the presence of a nonsense mutation in the gene or a premature stop codon in the mRNA, pharmaceutical formulation comprising said oxadiazole derivatives and prodrug or mixture thereof and the methods for the preparation of said Oxadiazole derivatives.

Evaluation and improvement of the quality of the discharge prescription in Cystic Fibrosis children hospitalised for intravenous antibiotic therapy

2017

1.Antecedentes. La Fibrosis Quística (FQ) es una enfermedad conocida desde la edad media, aunque durante esa época no estaba descrita como tal sino más bien como un hechizo maligno. En 1943, Farber propuso el término mucoviscidosis y este término se sigue utilizando en la actualidad. La FQ es la enfermedad hereditaria autosómica recesiva grave más frecuente en la población blanca, con una incidencia en Europa que oscila entre 1:1.353 en Irlanda y entre 1:25.000 en Finlandia, y una frecuencia de portadores de 1 por cada 2527. Es una enfermedad de las células epiteliales exocrinas, que se caracteriza por la producción de un moco espeso y viscoso que obstruye los conductos del órgano donde se …

Novel molecules for the readthrough of PTCs in biological model systems and in cystic fibrosis cells

2016