Search results for "Dosis"

Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome

2019

Abstract This cross-sectional analysis assessed the correlation between patient-reported outcomes (PROs) and clinical outcomes in 24 German patients with Morquio A. Clinical outcomes included 6-minute walk test (6MWT), 3-minute stair climb (3MSC) test, and joint range of motion as measures for endurance/mobility, forced vital capacity (FVC) and maximum voluntary ventilation (MVV) as measures for respiratory function, and height as an important manifestation. The PROs included the EuroQoL (EQ) 5D-5L (EQ5D-5L), to measure health-related QoL (HRQoL), and patients’ rating of their ability to walk, climb, or breathe. In adults, endurance and pulmonary function measures and height showed strong a…

Evaluation of the long-term treatment effects of idursulfase using statistical modelling: Data from the Hunter Outcome Survey (HOS)

2019

Treatment for mucopolysaccharidosis type II (MPS II Hunter syndrome) is available in the form of intravenous enzyme replacement therapy (ERT) with idursulfase (Shire, Lexington, MA, USA). This analysis used statistical modelling to evaluate the long-term treatment effects of idursulfase on selected clinical parameters based on data from HOS, a global, observational registry (Shire, Lexington, MA, USA). Mixed modelling was used to analyse data from male patients followed prospectively in HOS who had received idursulfase for 5-8 years and information available for two or more timepoints, of which one was pre-ERT. Data were excluded from patients with only pre-ERT information available, who ha…

BONE Sarcoidosis: A French Case Control Study

2018

International audience

Early detection of amyloid light-chain amyloidosis: a case report of oral primary manifestation

2018

Independent predictors of survival in primary systemic (Al), amyloidosis, including cardiac biomarkers and left ventricular strain imaging: an observ…

2010

BACKGROUND: The prognostic value of Doppler myocardial imaging, including myocardial velocity imaging, strain, and strain rate imaging, in patients with primary (AL) amyloidosis is uncertain. The aim of this longitudinal study was to identify independent predictors of survival, comparing clinical data, hematologic and cardiac biomarkers, and standard echocardiographic and Doppler myocardial imaging measures in a cohort of patients with AL amyloidosis. METHODS: A total of 249 consecutive patients with AL amyloidosis were prospectively enrolled. The primary end point was all-cause mortality, and during a median follow-up period of 18 months, 75 patients (30%) died. Clinical and electrocardiog…

Asymptomatic Amyloidosis at the Time of Diagnostic Bone Marrow Biopsy in Newly Diagnosed Patients with Multiple Myeloma and Smoldering Multiple Myelo…

2009

Abstract Abstract 2803 Poster Board II-779 Background. The rate of asymptomatic amyloidosis (asym-amyloidosis) detected in patients with newly diagnosed multiple myeloma (MM) or smoldering multiple myeloma (SMM) is unknown. This topic is significant because unrecognized AL may be associated with increased mortality may change the patient's management. The objective of the present investigation was to evaluate the number and clinical significance of asym-amyloidosis in MM and SM patients at the time of the diagnostic bone marrow (BM) biopsy for MM. Materials and Methods. The study population was selected from the Mayo Clinic Dysproteinemia database and consisted of consecutive patients with …

Determinación de anticuerpos anti-Anisakis y relación con las subpoblaciones linfocitarias αβ Y γδ en pacientes con Enfermedad de Crohn

2015

La enfermedad de Crohn (EC) es una enfermedad de etiopatogenia y fisiopatología compleja, que se desarrolla sobre una base de múltiples alteraciones inmunes, tanto de la inmunidad innata como de la inmunidad adquirida. Siguiendo nuestra línea de investigación en relación a la deficiencia de LTγδ en pacientes con enfermedad de Crohn, planteamos la hipótesis de trabajo de que parásitos como Anisakis simplex, ingerido frecuentemente mediante el consumo de pescado, podrían aprovechar este estado de inmunidad alterada en la enfermedad de Crohn para infectar con mayor frecuencia a estos pacientes que a la población sana. Hemos realizado un estudio prospectivo de casos y controles desde junio de 2…

Experimental Inhibition of Fibrillogenesis and Neurotoxicity by amyloid-beta (Aβ) and Other Disease-Related Peptides/Proteins by Plant Extracts and H…

2012

Amyloid-β (Aβ) fibrillogenesis and associated cyto/neurotoxicity are major pathological events and hallmarks in diseases such as Alzheimer’s disease (AD). The understanding of Aβ molecular pathogenesis is currently a pharmacological target for rational drug design and discovery based on reduction of Aβ generation, inhibition of Aβ fibrillogenesis and aggregation, enhancement of Aβ clearance and amelioration of associated cytotoxicity. Molecular mechanisms for other amyloidoses, such as transthyretin amyloidosis, AL-amyloidosis, as well as α-synuclein and prion protein are also pharmacological targets for current drug therapy, design and discovery. We report on natural herbal compounds and e…

Hypoxia-Induced Extracellular Acidosis Increases p-Glycoprotein Activity and Chemoresistance in Tumors in Vivo via p38 Signaling Pathway

2011

Due to inadequate perfusion, tumors develop hypoxia and extracellular acidosis. In vitro, this acidic environment (pH=6.6) has a strong impact on the acitvity of the p-glycoprotein (pGP) drug transporter responsible for multidrug resistance. This effect is most probably mediated via p38 and/or ERK1/2 signalling pathways. The aimof the studywas to analyzewhether these findings also play a role for chemosensitivity in solid growing tumors in vivo. Therefore, experimental R3327-AT1 tumors of the rat were exposed to an acidifying treatment leading to forced glycolysis. The intratumoral pO21 was determined polarographically and the extracellular pH was measured with needle electrodes. In additio…

Neuron-Glial Interaction During Injury and Edema of the CNS

1994

During injury and ischemia of the CNS mediator compounds are released or activated which cause secondary swelling and damage of nerve cells. Such mediators are glutamate, acidosis, free fatty acids, or high extracellular potassium. Glial homeostatic mechanisms are activated to prevent the secondary injury from these mediators. The glial clearance mechanisms have been studied in detail using in vitro systems allowing for a close control of the glial environment. Current evidence suggests glial swelling to occur together with glutamate uptake or in response to extracellular acidosis. Glial swelling, therefore, is rather the result of homeostatic mechanisms than an indication of glial demise.