Search results for "AOS"
showing 10 items of 330 documents
Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS).
2019
Fabry's disease (FD) is an X-linked lysosomal storage disorder caused by the deficient activity of the lysosomal enzyme alpha-galactosidase A (alpha-Gal A) leading to intracellular accumulation of globotriaosylceramide (Gb3). Patients with amenable mutations can be treated with migalastat, a recently approved oral pharmacologic chaperone to increase endogenous alpha-Gal A activity. We assessed safety along with cardiovascular, renal, and patient-reported outcomes and disease biomarkers in a prospective observational multicenter study after 12 months of migalastat treatment under real-world conditions. Fifty-nine (28 females) patients (34 (57.6%) pretreated with enzyme replacement therapy) w…
Fabry disease: overall effects of agalsidase alfa treatment
2004
Background Fabry disease is a rare X-linked disorder caused by deficient activity of the lysosomal enzyme α-galactosidase A. Progressive accumulation of the substrate globotriaosylceramide in cells throughout the body leads to major organ failure and premature death. The Fabry Outcome Survey (FOS) is a European outcomes database which was established to collect data on the natural history of this little-known disease and to monitor the long-term efficacy and safety of enzyme replacement therapy (ERT) with agalsidase alfa. This paper presents the first analysis of the FOS database on the effects of ERT on renal function, heart size, pain and quality of life. Design The effects of 1 and 2 y…
Changes in plasma and urine globotriaosylceramide levels do not predict Fabry disease progression over 1 year of agalsidase alfa.
2013
Globotriaosylceramide concentrations were assessed as potential predictors of change from baseline after 12 months by estimated glomerular filtration rate and left-ventricular mass index using pooled data from three randomized, placebo-controlled agalsidase alfa trials and open-label extensions of patients with Fabry disease.Males (aged 18 years or older) with Fabry disease received agalsidase alfa (0.2 mg/kg every other week for 12 months). A backward-elimination approach evaluated potential predictors (baseline estimated glomerular filtration rate and left-ventricular mass index; age at first dose; baseline and change from baseline at 12 months of globotriaosylceramide (urine, plasma); ur…
Histopathology of ameloblastoma of the jaws; some critical observations based on a 40 years single institution experience
2011
The aim of the present study is to examine all cases of intraosseous benign ameloblastomas treated between 1970 and 2010 in a single institution and to look for a possible correlation between the histopathological aspects and the demographical and clinical parameters, as well as the treatment outcome. The data of a total number of 44 patients were retrieved from the records. Nine patients were excluded because of doubt about the correct diagnosis (8 patients) or because of an extra-osseous presentation (1 patient). No statistically significant differences were found between the histopathological (sub)types of ameloblastomas and the demographical and clinical parameters, nor between the hist…
Uncertainty quantification in simulations of epidemics using polynomial chaos.
2012
Mathematical models based on ordinary differential equations are a useful tool to study the processes involved in epidemiology. Many models consider that the parameters are deterministic variables. But in practice, the transmission parameters present large variability and it is not possible to determine them exactly, and it is necessary to introduce randomness. In this paper, we present an application of the polynomial chaos approach to epidemiological mathematical models based on ordinary differential equations with random coefficients. Taking into account the variability of the transmission parameters of the model, this approach allows us to obtain an auxiliary system of differential equa…
Vēsture: Latvijas Universitātes Žurnāls, 2018, Nr.6
2019
Sources of geomaterials in the Sicani Mountains during the Early Middle Ages: A case study of Contrada Castro, central western Sicily
2022
From 2017, an unknown rural settlement in Contrada Castro at Corleone (Palermo Province, western Sicily) was investigated as part of the `Harvesting Memories Project¿. The stratigraphic sequence, supported by radiocarbon dating, has demonstrated a reoccupation of a pre-Roman site during the transition between the Byzantine and Islamic periods. In particular, the main occupation occurred in the late 8th¿9th century when pottery kilns and a probable warehouse were constructed. During the 10th¿11th century, a new structure with different orientations replaced the previous buildings that had already collapsed. Specifically focusing on a perspective of the household production and its relationsh…
Redécouverte d'une faune pléistocène dans les remplissages karstiques de Tam Hang au Laos : premiers résultats.
2008
12 pages; The discovery of a Pleistocene fauna in karstic fills at TamHang in Laos: preliminary results. TamHang, located in northeastern Laos, in the Hua Pan province, is a reference site not only for its numerous archaeological and human remains, but also for its two faunal assemblages, whose composition suggests different dating in the Pleistocene. Discovered in 1934 by Jacques Fromaget, a new excavation of the site has been undertaken in 2003. During this campaign, at Tam Hang south, 575 isolated teeth of mammals were extracted from a deep layer of calcareous breccia. This assemblage corresponds to that one described by Arambourg and Fromaget [C. Arambourg, J. Fromaget, Le gisement quat…
The onset of the rainy season and farmers’ sowing strategy for pearl millet cultivation in Southwest Niger
2011
A multi-year (2004-2009) field survey of on-farm sowing practices in 10 villages located in south-west Niger close to Niamey, is analysed to investigate the relationships (i) between rainfall and the sowing date of pearl millet and the risk of sowing failure, (ii) between sowing and meteorological/agro-climatic onset dates, (iii) between sowing/onset dates, and simulated and observed yield/biomass at the end of the season. Even if some villages sow without any synchronous or anterior rainfall, most parcels (73% out of the 1551 available cases) are sown during and just after a 2-day wet spell receiving at least 10 mm. In fact, there is a strong correlation (r = 0.82-0.95 depending on onset d…