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https://www.readbyqxmd.com/read/28208664/the-role-of-the-neutrophil-to-lymphocyte-ratio-for-survival-outcomes-in-patients-with-metastatic-castration-resistant-prostate-cancer-treated-with-abiraterone
#1
Martin Boegemann, Katrin Schlack, Stefan Thomes, Julie Steinestel, Kambiz Rahbar, Axel Semjonow, Andres Jan Schrader, Martin Aringer, Laura-Maria Krabbe
The purpose of this study was to examine the prognostic capability of baseline neutrophil-to-lymphocyte-ratio (NLR) and NLR-change under Abiraterone in metastatic castration-resistant prostate cancer patients. The impact of baseline NLR and change after eight weeks of treatment on progression-free survival (PFS) and overall survival (OS) was analyzed using Kaplan-Meier-estimates and Cox-regression. 79 men with baseline NLR <5 and 17 with NLR >5 were analyzed. In baseline analysis of PFS NLR >5 was associated with non-significantly shorter median PFS (five versus 10 months) (HR: 1...
February 11, 2017: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/28193891/mapping-evaporative-water-loss-in-desert-passerines-reveals-an-expanding-threat-of-lethal-dehydration
#2
Thomas P Albright, Denis Mutiibwa, Alexander R Gerson, Eric Krabbe Smith, William A Talbot, Jacqueline J O'Neill, Andrew E McKechnie, Blair O Wolf
Extreme high environmental temperatures produce a variety of consequences for wildlife, including mass die-offs. Heat waves are increasing in frequency, intensity, and extent, and are projected to increase further under climate change. However, the spatial and temporal dynamics of die-off risk are poorly understood. Here, we examine the effects of heat waves on evaporative water loss (EWL) and survival in five desert passerine birds across the southwestern United States using a combination of physiological data, mechanistically informed models, and hourly geospatial temperature data...
February 13, 2017: Proceedings of the National Academy of Sciences of the United States of America
https://www.readbyqxmd.com/read/28170189/mesenchymal-stem-cells-yield-transient-improvements-in-motor-function-in-an-infant-rhesus-macaque-with-severe-early-onset-krabbe-disease
#3
Irina A Isakova, Kate C Baker, Jason Dufour, Donald G Phinney
Krabbe disease, or globoid cell leukodystrophy, is a rare disorder caused by deficient galactosylceramidase activity and loss of myelin-forming oligodendrocytes, resulting in progressive demyelination and severely impaired motor function. Disease symptoms in humans appear within 3-6 months of age (early infantile) and manifest as marked irritability, spasticity, and seizures. The disease is often fatal by the second year of life, with few effective treatment options. Herein we evaluated the therapeutic potential of mesenchymal stem cells (MSCs) administered intracranially to a 1-month-old rhesus macaque diagnosed with severe early-onset Krabbe disease that displayed neurologic and behavioral symptoms similar to those of human patients...
January 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28168127/krabbe-disease-report-of-a-rare-lipid-storage-and-neurodegenerative-disorder
#4
Pratyusha Pavuluri, Sabitha Vadakedath, Rajkumar Gundu, Sushmitha Uppulety, Venkataramana Kandi
Krabbe disease is a rare (one in 100,000 births) autosomal recessive condition, usually noticed among children. It causes sphingolipidosis (dysfunctional metabolism of sphingolipids) and leads to fatal degenerative changes affecting the myelin sheath of the nervous system. We report a case of a six-year-old male child who presented with symptoms of muscle spasticity and irritability. Diagnosis of this disease can only be made with clinical suspicion. Laboratory diagnosis includes brain magnetic resonance imaging (MRI), magnetic resonance (MR) spectroscopy, biochemical analysis of cerebrospinal fluid, and genetic analysis for detecting mutation in genes coding for galactosyl cerebroside (GALC)...
January 1, 2017: Curēus
https://www.readbyqxmd.com/read/28145830/corrigendum
#5
(no author information available yet)
Hinderink JB, Krabbe PFM, van den Broek P. Development and application of a health-related quality-of-life instrument for adults with cochlear implants: The Nijmegen Cochlear Implant Questionnaire. Otolaryngol Head Neck Surg. 2000;123:756-765. (Original DOI: 10.1067/mhn.2000.108203 ).
February 2017: Otolaryngology—Head and Neck Surgery
https://www.readbyqxmd.com/read/28117439/a-competitive-inhibitory-circuit-for-selection-of-active-and-passive-fear-responses
#6
Jonathan P Fadok, Sabine Krabbe, Milica Markovic, Julien Courtin, Chun Xu, Lema Massi, Paolo Botta, Kristine Bylund, Christian Müller, Aleksandar Kovacevic, Philip Tovote, Andreas Lüthi
When faced with threat, the survival of an organism is contingent upon the selection of appropriate active or passive behavioural responses. Freezing is an evolutionarily conserved passive fear response that has been used extensively to study the neuronal mechanisms of fear and fear conditioning in rodents. However, rodents also exhibit active responses such as flight under natural conditions. The central amygdala (CEA) is a forebrain structure vital for the acquisition and expression of conditioned fear responses, and the role of specific neuronal sub-populations of the CEA in freezing behaviour is well-established...
January 25, 2017: Nature
https://www.readbyqxmd.com/read/28109651/late-onset-krabbe-disease-due-to-the-new-galc-p-ala543pro-mutation-with-intriguingly-high-residual-galc-activity-in%C3%A2-vitro
#7
Inge Krägeloh-Mann, Klaus Harzer, Kevin Rostásy, Stefanie Beck-Wödl, Antje Bornemann, Judith Böhringer, Andrea Bevot, Verena Beck, Gisela Merkel, Maria Pechan
BACKGROUND: Krabbe disease (KD) is an inherited leukodystrophy due to a defect in the GALC gene which encodes the lysosomal galactosylceramide β-galactosidase (GALC). About two thirds of patients show the early onset form of KD dominated by cerebral demyelination leading to death in early infancy. Late onset forms include a spectrum of late infantile, juvenile and adult clinical courses. The deficiency of GALC leads to a galactosylceramide lipidosis in which lysosomal storage phenomena are seen almost only at the ultrastructural level...
January 6, 2017: European Journal of Paediatric Neurology: EJPN
https://www.readbyqxmd.com/read/28103109/impaired-spermatogenesis-in-the-twitcher-mouse-a-morphological-evaluation-from-the-seminiferous-tubules-to-epididymal-transit
#8
Alice Luddi, Martina Gori, Laura Crifasi, Camilla Marrocco, Giuseppe Belmonte, Elvira Costantino-Ceccarini, Piomboni Paola
: Spermatogenesis is a complex process of proliferation and differentiation during male germ cell development whereby undifferentiated spermatogonial germ cells evolve into maturing spermatozoa. In this developmental process the interactions between different cell types are finely regulated, hence any disruption in these relationships leads to male infertility. The twitcher mouse, the murine model of Krabbe disease, is characterized by deficiency of galactosylceramidase, an enzyme also involved in the metabolism of the galactosyl-alkyl-acyl-glycerol, the precursor of sulfogalactosyl-alkyl-acyl-glycerol, the most abundant glycolipid in spermatozoa...
January 19, 2017: Systems Biology in Reproductive Medicine
https://www.readbyqxmd.com/read/28095876/multi-criteria-decision-analysis-mcda-testing-a-proposed-mcda-framework-for-orphan-drugs
#9
C Schey, P F M Krabbe, M J Postma, M P Connolly
BACKGROUND: Since the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. This study sought to explore the multi-criteria decision analysis (MCDA) framework proposed by (Orphanet J Rare Dis 7:74, 2012) to a range of orphan drugs, with a view to comparing the aggregate scores to the average annual cost per patient for each product, and thus establishing the merit of MCDA as a tool for assessing the value of orphan drugs in relation to their pricings...
January 17, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28075543/prognostic-value-of-tissue-based-biomarker-signature-in-clear-cell-renal-cell-carcinoma
#10
Ahmed Q Haddad, Jun-Hang Luo, Laura-Maria Krabbe, Oussama Darwish, Bishoy Gayed, Ramy Youssef, Payal Kapur, Dinesh Rakheja, Yair Lotan, Arthur Sagalowsky, Vitaly Margulis
OBJECTIVE: To improve risk stratification for recurrence prognostication in localized clear cell renal cell carcinoma (ccRCC) patients. PATIENTS AND METHODS: 367 patients with non-metastatic ccRCC were included. The cohort was divided into a training and validation set. Using tissue microarrays, immunostaining was performed for 24 biomarkers representative of key pathways in ccRCC. Using LASSO Cox regression, we identified several markers which were used to construct a risk classifier for risk of disease recurrence...
January 11, 2017: BJU International
https://www.readbyqxmd.com/read/28052886/intestinal-rehabilitation-for-children-with-intestinal-failure-is-cost-effective-a-simulation-study
#11
Henk Groen, Esther G Neelis, Marten J Poley, Joanne F Olieman, René Scheenstra, Paul Fm Krabbe, Gerard Dijkstra, Edmond Hhm Rings
BACKGROUND: Children with intestinal failure (IF) depend on parenteral nutrition (PN). The goal in the treatment of IF is to wean children off PN through intestinal rehabilitation (IR). Although the healthcare burden of IF is enormous, to our knowledge there has been no previous cost-effectiveness analysis in pediatric IF including IR. OBJECTIVE: We sought to determine the cost-effectiveness of IR in terms of costs and life-years. DESIGN: We simulated the treatment of IF in children in a discrete-event model...
January 4, 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28050682/competition-and-quality-indicators-in-the-health-care-sector-empirical-evidence-from-the-dutch-hospital-sector
#12
R R Croes, Y J F M Krabbe-Alkemade, M C Mikkers
There is much debate about the effect of competition in healthcare and especially the effect of competition on the quality of healthcare, although empirical evidence on this subject is mixed. The Netherlands provides an interesting case in this debate. The Dutch system could be characterized as a system involving managed competition and mandatory healthcare insurance. Information about the quality of care provided by hospitals has been publicly available since 2008. In this paper, we evaluate the relationship between quality scores for three diagnosis groups and the market power indicators of hospitals...
January 3, 2017: European Journal of Health Economics: HEPAC: Health Economics in Prevention and Care
https://www.readbyqxmd.com/read/28045982/fecal-bile-salts-and-the-development-of-necrotizing-enterocolitis-in-preterm-infants
#13
Christian V Hulzebos, Anne G J F van Zoonen, Jan B F Hulscher, Trijntje E Schat, Elisabeth M W Kooi, Martijn Koehorst, Renze Boverhof, Paul F M Krabbe, Albert K Groen, Henkjan J Verkade
BACKGROUND: Intestinal bile salts (BSs) may be implicated in NEC development. We hypothesized that fecal BS levels are higher in preterm infants at risk for NEC. METHODS: We compared the composition and concentration of fecal BSs in ten preterm infants who developed NEC (Bell's Stage ≥ II) with twenty matched control infants without NEC. Conjugated and unconjugated fecal BSs were measured after birth (T1) and twice prior to NEC (T2, T3). Data are presented as medians and interquartile ranges...
2017: PloS One
https://www.readbyqxmd.com/read/28000364/a-specific-activity-based-probe-to-monitor-family-gh59-galactosylceramidase-the-enzyme-deficient-in-krabbe-disease
#14
André R A Marques, Lianne I Willems, Daniela Herrera Moro, Bogdan I Florea, Saskia Scheij, Roelof Ottenhoff, Cindy P A A van Roomen, Marri Verhoek, Jessica K Nelson, Wouter W Kallemeijn, Anna Biela-Banas, Olivier R Martin, M Begoña Cachón-González, Nee Na Kim, Timothy M Cox, Rolf G Boot, Herman S Overkleeft, Johannes M F G Aerts
Galactosylceramidase (GALC) is the lysosomal β-galactosidase responsible for the hydrolysis of galactosylceramide. Inherited deficiency in GALC causes Krabbe disease, a devastating neurological disorder characterized by accumulation of galactosylceramide and its deacylated counterpart, the toxic sphingoid base galactosylsphingosine (psychosine). We report the design and application of a fluorescently tagged activity-based probe (ABP) for the sensitive and specific labeling of active GALC molecules from various species...
December 20, 2016: Chembiochem: a European Journal of Chemical Biology
https://www.readbyqxmd.com/read/27993207/gallbladder-abnormalities-in-children-with-metachromatic-leukodystrophy
#15
Jina Kim, Zhifei Sun, Brian Ezekian, Gary R Schooler, Vinod K Prasad, Joanne Kurtzberg, Henry E Rice, Elisabeth T Tracy
BACKGROUND: Metachromatic leukodystrophy (MLD) is a lysosomal storage disease that leads to neurological deterioration and visceral involvement, including sulphatide deposition in the gallbladder wall. Using our institution's extensive experience in treating MLD, we examined the incidence of gallbladder abnormalities in the largest cohort of children with MLD to date. METHODS: We conducted a retrospective review of all children with MLD, adrenoleukodystrophy (ALD), or Krabbe disease who underwent hematopoietic stem cell transplantation (HSCT) at our institution between 1994 and 2015...
February 2017: Journal of Surgical Research
https://www.readbyqxmd.com/read/27977664/lysosomal-re-acidification-prevents-lysosphingolipid-induced-lysosomal-impairment-and-cellular-toxicity
#16
Christopher J Folts, Nicole Scott-Hewitt, Christoph Pröschel, Margot Mayer-Pröschel, Mark Noble
Neurodegenerative lysosomal storage disorders (LSDs) are severe and untreatable, and mechanisms underlying cellular dysfunction are poorly understood. We found that toxic lipids relevant to three different LSDs disrupt multiple lysosomal and other cellular functions. Unbiased drug discovery revealed several structurally distinct protective compounds, approved for other uses, that prevent lysosomal and cellular toxicities of these lipids. Toxic lipids and protective agents show unexpected convergence on control of lysosomal pH and re-acidification as a critical component of toxicity and protection...
December 2016: PLoS Biology
https://www.readbyqxmd.com/read/27973216/translation-adaptation-and-validation-of-the-diabetes-distress-scale-for-indonesian-diabetic-outpatients-with-various-types-of-complications
#17
B Arifin, D Perwitasari, J Atthobari, Q Cao, P F Krabbe, M J Postma
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27920424/ultrastructural-characterization-of-the-lower-motor-system-in-a-mouse-model-of-krabbe-disease
#18
Valentina Cappello, Laura Marchetti, Paola Parlanti, Silvia Landi, Ilaria Tonazzini, Marco Cecchini, Vincenzo Piazza, Mauro Gemmi
Krabbe disease (KD) is a neurodegenerative disorder caused by the lack of β- galactosylceramidase enzymatic activity and by widespread accumulation of the cytotoxic galactosyl-sphingosine in neuronal, myelinating and endothelial cells. Despite the wide use of Twitcher mice as experimental model for KD, the ultrastructure of this model is partial and mainly addressing peripheral nerves. More details are requested to elucidate the basis of the motor defects, which are the first to appear during KD onset. Here we use transmission electron microscopy (TEM) to focus on the alterations produced by KD in the lower motor system at postnatal day 15 (P15), a nearly asymptomatic stage, and in the juvenile P30 mouse...
December 2016: Scientific Reports
https://www.readbyqxmd.com/read/27887956/-childhood-s-ophthalmologic-involvement-in-sturge-weber-krabbe-syndrome
#19
M Wirth, M-C Bazard, E Schmitt, M Rouabah, J-M Hascoët
Ophthalmologic involvement in Sturge-Weber-Krabbe syndrome (SWKS) is present in 30-70% of cases and needs to be reviewed because of its impact on visual development. We report a case of a newborn for whom SSWK with ophthalmologic involvement was suspected. She had a right segmental plane angioma associated with right congenital glaucoma and suspected right pial angioma. Ophthalmic involvement in SWKS may be nonocular: iatrogenic by treatment-associated complications and central by leptomeningeal damage. Ophthalmologic involvement can occur throughout childhood and is mainly represented by glaucoma and diffuse choroidal hemangioma and then conjunctival hemangioma, retinal detachment, and iris heterochromia...
January 2017: Archives de Pédiatrie: Organe Officiel de la Sociéte Française de Pédiatrie
https://www.readbyqxmd.com/read/27807631/head-to-head-comparison-of-health-state-values-derived-by-a-probabilistic-choice-model-and-scores-on-a-visual-analogue-scale
#20
Paul F M Krabbe, Elly A Stolk, Nancy J Devlin, Feng Xie, Elise H Quik, A Simon Pickard
BACKGROUND: Health states were quantified based on discrete choice (DC) modeling and visual analogue scale (VAS) values using the five-level version of the EQ-5D (EQ-5D-5L). The aim of this study was to determine the extent of the relationship between DC derived values (indirect method) and VAS values (direct method). METHODS: Data were collected in Canada, the United Kingdom, the Netherlands, and the United States. Respondents were asked to perform paired comparisons between two EQ-5D-5L health states for DC...
November 2, 2016: European Journal of Health Economics: HEPAC: Health Economics in Prevention and Care
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