Search results for "cystic fibrosi"
showing 10 items of 115 documents
Liver disease as risk factor for cystic fibrosis-related diabetes development
2007
Aim: To evaluate clinical and genetic factors, besides pancreatic insufficiency, associated with increased risk of cystic fibrosis-related diabetes. Methods: Case-control (1:1) study on 138 cystic fibrosis patients. Data were collected on gender, age at diagnosis, reason for cystic fibrosis diagnosis, family history of type 1 or 2 diabetes mellitus, pre-existing severe liver disease, and class of cystic fibrosis transmembrane regulation mutation. Moreover, information was obtained on lung involvement and degree of exocrine pancreatic insufficiency evaluated 1 year before the diagnosis of cystic fibrosis-related diabetes in patients and age-matched controls. Results: Compared to controls, pa…
Strategies for Survival of Extracellular Pathogens
2002
Publisher Summary This chapter highlights a simple listing of microbial factors that may contribute to extracellular survival. In addition, it outlines principles and discusses prototypes for extracellular versus intracellular survival strategies. Strategies for extracellular survival can often be equated with pathogenicity mechanisms, and counteractive measures can therefore form the basis for prophylaxis or therapy of the respective infections. These aspects are aluded throughout the chapter, since they investigates how basic research into microbial virulence mechanisms can lead to advances in clinical medicine. Most bacteria that cause extracellular infections cannot afford to be engulfe…
The CFTR associated protein CAP70 interacts with the apical Cl-/HCO3- exchanger DRA in rabbit small intestinal mucosa.
2005
DRA (down regulated in adenoma) is an intestinal anion exchanger, acting in parallel with NHE3 to facilitate ileal and colonic NaCl absorption. Furthermore it is involved in small intestinal bicarbonate secretion. Because DRA has a PDZ interaction motif, which may influence its properties, we searched for DRA-interacting PDZ adapter proteins in the small intestine. Using an overlay assay with the recombinant DRA C-terminus as a ligand, a 70 kDa protein was labeled, which was restricted to the brush border membrane in rabbit duodenal and ileal mucosa and was not detected in the colon. Destruction of the C-terminal PDZ interaction motif abolished this band, suggesting a specific protein-prote…
Inhalation solutions: which one are allowed to be mixed? Physico-chemical compatibility of drug solutions in nebulizers.
2006
AbstractTherapy of chronic respiratory diseases often involves inhalation therapy with nebulizers. Patients often attempt to shorten the time consuming administration procedure by mixing drug solutions/suspensions for simultaneous inhalation. This article considers the issue of physico-chemical compatibility of admixtures of drug solutions/suspensions in nebulizers.A search of databases, prescribing information and primary literature was conducted to locate literature concerning the physico-chemical compatibility of inhalation solutions/suspensions. This was supplemented by telephone interviews.Admixtures of albuterol with ipratropium and/or cromolyn, of albuterol and budesonide, or tobramy…
Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the read-through of PTCs in CF …
2015
The role of attachment patterns and family functioning on psychophysical well-being in children with Cystic Fibrosis Preliminary Results
2013
(1) Introduction Scientific literature evidences a link between the quality of personal relationships and health status: the attachment style has a role on health and growth outcomes (Feeney, 2000) especially in chronic illness representing a threatening situation (Ciechanowski, 2003). Cystic fibrosis (CF) is a chronic genetic condition compromising the exocrine glands functioning and requiring intensive daily therapies (White et al., 2009). In pediatric condition, infant-mother relationship constituted an important element for nutritional status in CF children (Simmons et al., 1995; Anthony et al. 1999) which is one of the major factors contributing to improve longevity and quality of life…
Furocoumarins as multi-target agents in the treatment of cystic fibrosis.
2019
Multi-target molecular entities, offer a path to progress both in understanding causes of disease and in defining effective small molecule treatments. Coumarin and its derivatives belong to an important group of natural compounds with diverse biological properties. They are found in vegetables and plants for which literature reports thousands of publications for the great variety of biological applications among which the photoprotective effects, thus being considered multi-targeting agents. Their furan condensed analogues constitute the family of furocoumarins, less represented in the literature, endowed with photosensitizing properties and often used for the treatment of skin diseases suc…
Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives.
2015
Abstract Premature stop codons are the result of nonsense mutations occurring within the coding sequence of a gene. These mutations lead to the synthesis of a truncated protein and are responsible for several genetic diseases. A potential pharmacological approach to treat these diseases is to promote the translational readthrough of premature stop codons by small molecules aiming to restore the full-length protein. The compound PTC124 (Ataluren) was reported to promote the readthrough of the premature UGA stop codon, although its activity was questioned. The potential interaction of PTC124 with mutated mRNA was recently suggested by molecular dynamics (MD) studies highlighting the importanc…
Inhalable nano into micro dry powders for ivacaftor delivery: The role of mannitol and cysteamine as mucus-active agents.
2020
In this paper the innovative approach of Nano into micro (NiM9 was developed to produce Nanoparticles loaded Ivacaftor to incorporate into mannitol or mannitol/cysteamine micromatrices for drug pulmonary administration in CF. Nanoparticles composed by a mixture of two polyhydrohydroxyethtylaspartamide copolymers containing a loading of Ivacaftor of 15.5 % w/w were produced. These Nanoparticles were incorporated into microparticles to obtain NiM that were characterized in terms of size and size distribution, interaction with CF-AM by rheological and turbidimetric studies as well as by aerodynamic diameter measurements. Finally the activity of Ivacaftor into these NiM was evaluated by in vitr…
HETEROCYCLIC COMPOUNDS AND MEDICAL USE THEREOF
2019
The present invention relates to heterocyclic nitrogen compounds, use thereof as a medicament and pharmaceutical compositions thereof. Furthermore, the invention provides combinations of compounds of general formula (I) with therapeutic agents, such as correctors, potentiators and amplifiers of dysfunctional proteins.