Search results for "Fase"
showing 10 items of 94 documents
The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
2017
Mucopolysaccharidosis (MPS) VI is an autosomal recessive lysosomal storage disorder arising from deficient activity of N-acetylgalactosamine-4-sulfatase (arylsulfatase B) and subsequent intracellular accumulation of the glycosaminoglycans (GAGs) dermatan sulfate and chondroitin-4-sulfate. Manifestations are multi-systemic and include skeletal abnormalities such as dysostosis multiplex and short stature. Reference height-for-age growth charts for treatment-naive MPS VI patients have been published for both the slowly and rapidly progressing populations. Categorization of disease progression for these charts was based on urinary GAG (uGAG) level; high (>200μg/mg creatinine) levels identified …
Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study.
2017
Efficacy and safety of elosulfase alfa enzyme replacement therapy (ERT) were assessed in an open-label, phase 2, multi-national study in Morquio A patients aged ≥5 years unable to walk ≥30 meters in the 6-min walk test. Patients received elosulfase alfa 2.0 mg/kg/week intravenously for 48 weeks. Efficacy measures were functional dexterity, pinch/grip strength, mobility in a modified timed 25-foot walk, pain, quality of life, respiratory function, and urine keratan sulfate (KS). Safety/tolerability was also assessed. Fifteen patients received elosulfase alfa, three patients discontinued ERT due to adverse events (two were grade 3 drug-related adverse events, the other was not drug-related), …
Danger zone analysis using cone beam computed tomography after apical enlargement with K3 and K3XF in a manikin model
2015
Background The objective of the study was to evaluate and compare how apical enlargement with K3 and K3XF nickel-titanium (NiTi) rotary instruments reduces the root thickness in the danger zone and affects canal transportation and centering ability in mandibular molar mesial canals in a manikin extracted tooth model. Material and Methods Seventy-two mesial root canals of first mandibular molars were instrumented. Initial and post-instrumentation Cone Beam Computed Tomography scans were performed after root canal preparation up to size 25, 30, 35 and 40 files. Canal transportation, canal centering and remaining root dentin thickness toward the danger zone were calculated in sections 1, 2 and…
Sub-analysis of long-term elosulfase alfa treatment outcomes in adults with Morquio syndrome type A
2017
Emisiones difusas: medidas experimentales y modelización aplicadas a instalaciones de tratamiento de aguas residuales
2015
Los olores emitidos por la actividad rutinaria de una planta de tratamiento de aguas residuales (EDAR) representan una importante fuente de molestia medioambiental que, por lo general, implica la liberación atmosférica de sustancias que causan mal olor y problemas de salud asociados. Actualmente, existe una preocupación pública sobre los olores que se generan con trabajos de tratamiento de residuos. Esta preocupación ha aumentado en la última década debido principalmente a la sobrecarga en las zonas de captación y la invasión de núcleos habitados en torno a instalaciones de tratamiento de aguas residuales (reduciendo los márgenes de seguridad impuestos a éstos). Estas potenciales emisiones …
Aislamiento de cocaína y benzoilecgonina en muestras de orina por extracciones líquido-líquido y en fase sólida y confirmación por Cromatografía líqu…
2006
El consumo ilícito de cocaína se ha incrementado extraordinariamente en los últimos años, por lo que resulta indispensable el desarrollo de metodologías seguras, rápidas y eficientes para su detección. En este trabajo se desarrollo una técnica de HPLC de fase reversa con detector de UV para identificar y cuantificar a la cocaína y la benzoilecgonina, con resultados satisfactorios en los parámetros del control de calidad.Se realizó un estudio de recobrado para el aislamiento de la cocaína y la benzoilecgonina en orina con extracciones liquido liquido y en fase sólida con tres tipos de columnas comerciales (Bond Elut Certify, Extrelut 3 y Supelclean LC-18). Las fracciones obtenidas con la ext…
Estudio de caso clínico: diagnóstico dual
2010
El objetivo concreto de nuestro estudio era observar la evolución de los hábitos tóxicos de una paciente con patología dual y, el patrón de consumo en función de su patología mental (trastorno ciclotímico) y de estresores psicosociales. Así mismo esclarecimos los estadios en los que se encontraba y el proceso de cambio que estaba utilizando. También evaluamos su personalidad y su nivel de funcionamiento actual. Todo ello, con el objetivo principal de comprender mejor la comorbilidad entre los trastornos bipolares y los trastornos relacionados con sustancias. La paciente estudiada estaba en seguimiento en la UCA (Unidad de Conductas Adictivas) de Ciudad Real. Para dicho estudio utilizamos la…
Micelle in fase gas: una nuova frontiera della spettroMetria di Massa
2013
The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects
2013
Abstract Objectives The objectives of this study are to quantify endurance and respiratory function and better characterize spectrum of symptoms and biochemical abnormalities in mucopolysaccharidosis IVA subjects. Methods MorCAP was a multicenter, multinational, cross sectional study amended to be longitudinal in 2011. Each study visit required collection of medical history, clinical assessments, and keratan sulfate (KS) levels. Results Data from the first visit of 325 subjects (53% female) were available. Mean age was 14.5 years. Mean ± SD height z-scores were − 5.6 ± 3.1 as determined by the CDC growth charts. Mean ± SD from the 6-minute-walk-test was 212.6 ± 152.2 m, revealing limitation…
Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome
2015
Objectives: Baseline data from the Morquio A Clinical Assessment Program (MorCAP) revealed that individuals with Morquio A syndrome show substantial impairment in multiple domains including endurance and respiratory function (Harmatz et al., Mol Genet Metab, 2013). Here, 1- and 2-year longitudinal endurance and respiratory function data are presented.Methods: Endurance was assessed using the 6-minute walk test (6MWT) and the 3-minute stair climb test (3MSCT). Respiratory function was evaluated by measuring forced vital capacity (PVC) and maximum voluntary ventilation (MW). Data were analyzed using repeated measures ANCOVA models. Annualized estimates of change were determined using model es…