keyword
MENU ▼
Read by QxMD icon Read
search

ERT

keyword
https://www.readbyqxmd.com/read/28545984/efficacy-and-safety-of-de-novo-and-early-use-of-extended-release-tacrolimus-in-heart-transplantation
#1
Francisco González-Vílchez, José Luis Lambert, Diego Rangel, Luis Almenar, José Luis de la Fuente, Jesús Palomo, Beatriz Díaz Molina, Ernesto Lage, Ignacio Sánchez Lázaro, José A Vázquez de Prada
INTRODUCTION AND OBJECTIVES: The extended-release formulation of tacrolimus (ERT) allows once-daily dosage, thus simplifying the immunosuppressive regimen. This study aimed to describe the safety and efficacy of the de novo and early use of ERT in heart transplantation. METHODS: This was an observational, retrospective, multicenter study comparing the safety and efficacy of the de novo use of ERT (ERT group [n=94]), standard-release tacrolimus (SRT group [n=42]) and early conversion (EC) from SRT to ERT (EC group [n=44])...
May 22, 2017: Revista Española de Cardiología
https://www.readbyqxmd.com/read/28545342/echocardiographic-and-clinical-findings-in-patients-with-fabry-disease-during-long-term-enzyme-replacement-therapy-a-nationwide-danish-cohort-study
#2
Christoffer Valdorff Madsen, Henning Bundgaard, Åse Krogh Rasmussen, Søren Schwartz Sørensen, Jørgen Holm Petersen, Lars Køber, Ulla Feldt-Rasmussen, Helle Petri
OBJECTIVES: In patients with Fabry disease (FD), left ventricular hypertrophy and arrhythmias are frequently observed and cardiac involvement is the leading cause of death. Long-term efficacy of enzyme replacement therapy (ERT) on cardiac involvement is unclear. We assessed and compared long-term progression of cardiac involvement according to ERT and non-ERT. METHODS: We retrospectively assessed and compared long-term progression of cardiac involvement in adult patients with FD in the nationwide Danish cohort...
May 25, 2017: Scandinavian Cardiovascular Journal: SCJ
https://www.readbyqxmd.com/read/28540187/case-report-of-treatment-experience-with-idursulfase-beta-hunterase-in-an-adolescent-patient-with-mps-ii
#3
Lock-Hock Ngu, Winnie Ong Peitee, Huey Yin Leong, Hui Bein Chew
Mucopolysaccharidosis (MPS) II or Hunter syndrome is a chronic, progressive, multi-systemic illness associated with significant morbidity and early mortality. Available evidence in Asian populations shows that Hunter syndrome has a mean age of onset of 2 to 5 years and a life expectancy of 13 years in more severely affected individuals, with respiratory failure reported as the leading cause of death. Enzyme replacement therapy (ERT) with idursulfase (Elaprase, Shire Pharmaceuticals) and idursulfase beta (Hunterase, Green Cross Corp) are the only approved treatment for patients with MPS II...
September 2017: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/28535791/clinical-outcomes-in-a-subpopulation-of-adults-with-morquio-a-syndrome-results-from-a-long-term-extension-study-of-elosulfase-alfa
#4
D Hughes, R Giugliani, N Guffon, S A Jones, K E Mengel, R Parini, R Matousek, S M Hawley, A Quartel
BACKGROUND: This post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 years of age evaluated in an open-label, long-term extension study of elosulfase alfa (modified per protocol [MPP], n = 32; intent-to-treat [ITT], n = 37; MOR-005; NCT01415427) were compared with the ≥18-year-old untreated population with 2-years follow-up from a Morquio A natural history study (n = 10; MorCAP; NCT00787995)...
May 23, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28532341/pterostilbene-protects-against-uvb-induced-photo-damage-through-a-phosphatidylinositol-3-kinase-dependent-nrf2-are-pathway-in-human-keratinocytes
#5
Huaping Li, Na Jiang, Bihua Liang, Qing Liu, Erting Zhang, Liqian Peng, Huiyan Deng, Runxiang Li, Zhenjie Li, Huilan Zhu
OBJECTIVE: Ultraviolet B (UVB) irradiation is the initial etiological factor for various skin disorders, including erythema, sunburn, photoaging, and photocarcinogenesis. Pterostilbene (Pter) displayed remarkable antioxidant, anti-inflammatory, and anticarcinogenic activities. This study aimed to investigate the effective mechanism of Pter against UVB-induced photodamage in immortalized human keratinocytes. METHODS: Human keratinocytes were pretreated with Pter (5 and 10 μM) for 24 h prior to UVB irradiation (300 mJ/cm(2))...
May 22, 2017: Redox Report: Communications in Free Radical Research
https://www.readbyqxmd.com/read/28529638/cxcr4-directed-endoradiotherapy-induces-high-response-rates-in-extramedullary-relapsed-multiple-myeloma
#6
Constantin Lapa, Ken Herrmann, Andreas Schirbel, Heribert Hänscheid, Katharina Lückerath, Margret Schottelius, Malte Kircher, Rudolf A Werner, Martin Schreder, Samuel Samnick, Saskia Kropf, Stefan Knop, Andreas K Buck, Hermann Einsele, Hans-Juergen Wester, K Martin Kortüm
C-X-C-motif chemokine receptor 4 (CXCR4) is a key factor for tumor growth and metastasis in several types of human cancer. We have recently reported promising first-in-man experience with CXCR4-directed endoradiotherapy (ERT) in multiple myeloma (MM). Eight heavily pretreated MM patients underwent a total of 10 ERT cycles (7 patients with 1 cycle and a single patient with 3 cycles). ERT was administered in combination with chemotherapy and autologous stem cell support. End points were occurrence and timing of adverse events, progression-free and overall survival...
2017: Theranostics
https://www.readbyqxmd.com/read/28510034/improvement-of-fabry-disease-related-gastrointestinal-symptoms-in-a-significant-proportion-of-female-patients-treated-with-agalsidase-beta-data-from-the-fabry-registry
#7
William R Wilcox, Ulla Feldt-Rasmussen, Ana Maria Martins, Alberto Ortiz, Roberta M Lemay, Ana Jovanovic, Dominique P Germain, Carmen Varas, Katherine Nicholls, Frank Weidemann, Robert J Hopkin
Fabry disease, an X-linked inherited lysosomal storage disorder, is caused by mutations in the gene encoding α-galactosidase, GLA. In patients with Fabry disease, glycosphingolipids accumulate in various cell types, triggering a range of cellular and tissue responses that result in a wide spectrum of organ involvement. Although variable, gastrointestinal symptoms are among the most common and significant early clinical manifestations; they tend to persist into adulthood if left untreated. To further understand the effects of sustained enzyme replacement therapy (ERT) with agalsidase beta on gastrointestinal symptoms in heterozygotes, a data analysis of female patients enrolled in the Fabry Registry was conducted...
May 17, 2017: JIMD Reports
https://www.readbyqxmd.com/read/28507907/effectiveness-of-enzyme-replacement-therapy-in-fabry-disease-long-term-experience-in-argentina
#8
Gustavo Cabrera, Juan Politei, Norberto Antongiovani, Hernán Amartino
Evidence regarding long term effectiveness of enzyme replacement therapy (ERT) in Fabry disease (FD) is needed. The aim of this study was to analyze in a cohort of FD patients in Argentina, the long term effectiveness of ERT on renal, cardiac and cerebrovascular parameters. METHODS: Patients with genetically proven FD were included from GADYTEF (Argentinean group for the treatment of FD) between 2001 and 2014. Renal, cardiac, and cerebral outcomes were prospectively studied in patients treated with ERT...
June 2017: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/28506293/case-report-of-unexpected-gastrointestinal-involvement-in-type-1-gaucher-disease-comparison-of-eliglustat-tartrate-treatment-and-enzyme-replacement-therapy
#9
Yoo-Mi Kim, Dong Hoon Shin, Su Bum Park, Chong Kun Cheon, Han-Wook Yoo
BACKGROUND: Gastrointestinal involvement in Gaucher disease is very rare, and appears to be unresponsive to enzyme replacement therapy (ERT). CASE PRESENTATION: Here, we describe identical twin, splenectomized, non-neuronopathic Gaucher patients on long-term ERT for 9 years, who complained of epigastric discomfort due to Gaucher cell infiltration of the gastroduodenal mucosa. Rare compound heterozygous mutations (p.Arg48Trp and p.Arg257Gln) of the GBA gene were found in both...
May 15, 2017: BMC Medical Genetics
https://www.readbyqxmd.com/read/28502768/lysosomal-enzyme-replacement-therapies-historical-development-clinical-outcomes-and-future-perspectives
#10
Melani Solomon, Silvia Muro
Lysosomes and lysosomal enzymes play a central role in numerous cellular processes, including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies of lysosomal components, most commonly enzymes, are known as "lysosomal storage disorders" or "lysosomal diseases" (LDs) and lead to lysosomal dysfunction. LDs broadly affect peripheral organs and the central nervous system (CNS), debilitating patients and frequently causing fatality. Among other approaches, enzyme replacement therapy (ERT) has advanced to the clinic and represents a beneficial strategy for 8 out of the 50-60 known LDs...
May 11, 2017: Advanced Drug Delivery Reviews
https://www.readbyqxmd.com/read/28500230/conjunctival-lymphangiectasia-associated-with-classic-fabry-disease
#11
Melanie D Sivley, Eric L Wallace, David G Warnock, William J Benjamin
BACKGROUND: Fabry disease (FD) is a treatable multisystem disease caused by a defect in the alpha-galactosidase gene. Ocular signs of FD, including corneal verticillata, are among the earliest diagnostic findings. Conjunctival lymphangiectasia (CL) has not previously been associated with FD. METHODS: We examined the eyes of a cohort of 13 adult patients, eight men and five women, with documented classic FD, all treated with enzyme replacement therapy (ERT) at the University of Alabama at Birmingham between February 2014 and April 2015...
May 12, 2017: British Journal of Ophthalmology
https://www.readbyqxmd.com/read/28495078/favourable-effect-of-early-versus-late-start-of-enzyme-replacement-therapy-on-plasma-globotriaosylsphingosine-levels-in-men-with-classical-fabry-disease
#12
Maarten Arends, Frits A Wijburg, Christoph Wanner, Frédéric M Vaz, André B P van Kuilenburg, Derralynn A Hughes, Marieke Biegstraaten, Atul Mehta, Carla E M Hollak, Mirjam Langeveld
BACKGROUND: The level of plasma globotriaosylsphingosine (lysoGb3) is an indication of disease severity in Fabry disease (FD) and its decrease during enzyme replacement therapy could be a reflection of treatment efficacy. Early treatment of FD may improve clinical outcome, but data to support this hypothesis are scarce. In this study we compared lysoGb3 decrease after ERT initiation in men with classical FD who started ERT before the age of 25 (early-treatment) with those who started later in life (late-treatment)...
May 4, 2017: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/28491792/flecainide-treats-a-novel-kcnj2-mutation-associated-with-andersen-tawil-syndrome
#13
Hanora A Van Ert, Elise C McCune, Kate M Orland, Kathleen R Maginot, Nicholas H Von Bergen, Craig T January, Lee L Eckhardt
No abstract text is available yet for this article.
February 2017: HeartRhythm Case Reports
https://www.readbyqxmd.com/read/28487826/oxidative-profile-exhibited-by-mucopolysaccharidosis-type-iva-patients-at-diagnosis-increased-keratan-urinary-levels
#14
Bruna Donida, Desirèe P Marchetti, Carlos Eduardo Diaz Jacques, Graziela Ribas, Marion Deon, Paula Manini, Helen Tais da Rosa, Dinara Jaqueline Moura, Jenifer Saffi, Roberto Giugliani, Carmen Regla Vargas
Morquio A disease (Mucopolysaccharidosis type IVA, MPS IVA) is one of the 11 mucopolysaccharidoses (MPSs), a heterogeneous group of inherited lysosomal storage disorders (LSDs) caused by deficiency in enzymes need to degrade glycosaminoglycans (GAGs). Morquio A is characterized by a decrease in N-acetylgalactosamine-6-sulfatase activity and subsequent accumulation of keratan sulfate and chondroitin 6-sulfate in cells and body fluids. As the pathophysiology of this LSD is not completely understood and considering the previous results of our group concerning oxidative stress in Morquio A patients receiving enzyme replacement therapy (ERT), the aim of this study was to investigate oxidative stress parameters in Morquio A patients at diagnosis...
June 2017: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/28480166/albuterol-as-an-adjunctive-treatment-to-enzyme-replacement-therapy-in-infantile-onset-pompe-disease
#15
Yin-Hsiu Chien, Wuh-Liang Hwu, Ni-Chung Lee, Fuu-Jen Tsai, Dwight D Koeberl, Wen-Hui Tsai, Pao-Chin Chiu, Chaw-Liang Chang
BACKGROUND: Early initiation of enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase is an effective treatment for patients with infantile-onset Pompe disease (IOPD) but cannot prevent a slow progression of myopathy. Albuterol has been shown to be helpful in adult patients with Pompe disease, and therefore, we administered an open-label adjunctive therapy with albuterol in IOPD patients undergoing ERT. METHODS: Fourteen patients, aged 2 to 12 years, were enrolled in this study; all of them had a disease onset before 12 months of life, and 13 of them were ambulatory because of early initiation of ERT...
June 2017: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/28479223/the-frequency-of-carpal-tunnel-syndrome-in-hurler-syndrome-after-peritransplant-enzyme-replacement-therapy-a-retrospective-comparison
#16
Mitchell L Wyffels, Paul J Orchard, Ryan M Shanley, Weston P Miller, Ann E Van Heest
PURPOSE: Children with Hurler syndrome (HS) develop carpal tunnel syndrome (CTS) owing to glycosaminoglycan deposition secondary to enzyme deficiency. Advancement in the treatment of the underlying enzyme deficiency now commonly includes peritransplant intravenous enzyme replacement therapy (ERT). The primary objective of this study was to determine if the use of limited ERT in addition to hematopoietic stem cell transplantation (HCT) for the treatment of children with HS reduces the incidence of surgical intervention for CTS compared with a cohort of historical controls treated with HCT alone...
May 4, 2017: Journal of Hand Surgery
https://www.readbyqxmd.com/read/28477382/european-consensus-for-starting-and-stopping-enzyme-replacement-therapy-in-adult-patients-with-pompe-disease-a-10-year-experience
#17
A T van der Ploeg, M E Kruijshaar, A Toscano, P Laforêt, C Angelini, R H Lachmann, S I Pascual Pascual, M Roberts, K Rösler, T Stulnig, P A van Doorn, P Y K Van den Bergh, J Vissing, B Schoser
BACKGROUND AND PURPOSE: Pompe disease is a rare inheritable muscle disorder for which enzyme replacement therapy (ERT) has been available since 2006. Uniform criteria for starting and stopping ERT in adult patients were developed and reported here. METHODS: Three consensus meetings were organized through the European Pompe Consortium, a network of experts from 11 European countries in the field of Pompe disease. A systematic review of the literature was undertaken to determine the effectiveness of ERT in adult patients on a range of clinical outcome measures and quality of life...
June 2017: European Journal of Neurology: the Official Journal of the European Federation of Neurological Societies
https://www.readbyqxmd.com/read/28470334/transrectal-ultrasound-guided-prostate-biopsy-in-the-era-of-increasing-fluoroquinolone-resistance-prophylaxis-with-single-dose-ertapenem
#18
Michael Seitz, Christian Stief, Raphaela Waidelich, Markus Bader, Derya Tilki
PURPOSE: The aim of the study was to compare single-dose ertapenem (ERT) with the 3-day regime of ciprofloxacin (CIP) for prophylaxis of possible infections following transrectal prostate biopsy. METHODS: Data from a consecutive group of 542 patients from January 2012 to January 2017 were retrospectively analysed. As preinterventional prophylaxis patient group A (179) received 500 mg CIP twice a day for three days, beginning on the day before the biopsy (until June 2013); group B (363) received a single dose of ERT 60 min prior to intervention...
May 3, 2017: World Journal of Urology
https://www.readbyqxmd.com/read/28468960/genetic-disruption-of-adenosine-kinase-in-mouse-pancreatic-%C3%AE-cells-protects-against-high-fat-diet-induced-glucose-intolerance
#19
Guadalupe Navarro, Yassan Abdolazami, Zhengshan Zhao, Haixia Xu, Sooyeon Lee, Neali A Armstrong, Justin P Annes
Islet β-cells adapt to insulin resistance through increased insulin secretion and expansion. Type 2 diabetes (T2D) typically occurs when prolonged insulin resistance exceeds the adaptive capacity of β-cells. Our prior screening efforts led to the discovery that Adenosine kinase (ADK) inhibitors stimulate β-cell replication. Here evaluated whether ADK disruption in mouse β-cells impacts β-cell mass and/or protects against high fat diet (HFD)-induced glucose dysregulation. Mice targeted at the Adk locus were bred to Rip-Cre and Ins1-Cre/ERT(1Lphi) mice to enable constitutive (βADKO) and conditional (iβADKO) disruption of ADK expression in β-cells, respectively...
May 3, 2017: Diabetes
https://www.readbyqxmd.com/read/28468677/diagnosis-features-of-pediatric-gaucher-disease-patients-in-the-era-of-enzymatic-therapy-a-national-base-study-from-the-spanish-registry-of-gaucher-disease
#20
Marcio Andrade-Campos, Pilar Alfonso, Pilar Irun, Judith Armstrong, Carmen Calvo, Jaime Dalmau, Maria-Rosario Domingo, Jose-Luis Barbera, Horacio Cano, Maria-Angeles Fernandez-Galán, Rafael Franco, Inmaculada Gracia, Miguel Gracia-Antequera, Angela Ibañez, Francisco Lendinez, Marcos Madruga, Elena Martin-Hernández, Maria Del Mar O'Callaghan, Alberto Pérez Del Soto, Yolanda Ruiz Del Prado, Ignacio Sancho-Val, Pablo Sanjurjo, Miguel Pocovi, Pilar Giraldo
BACKGROUND: The enzymatic replacement therapy (ERT) availability for Gaucher disease (GD) has changed the landscape of the disease, several countries have screening programs. These actions have promoted the early diagnosis and avoided many complications in pediatric patients. In Spain ERT has been available since 1993 and 386 patients have been included in the Spanish Registry of Gaucher Disease (SpRGD). The aim of this study is to analyze the impact of ERT on the characteristics at time of diagnosis and initial complications in pediatric Gaucher disease patients...
May 3, 2017: Orphanet Journal of Rare Diseases
keyword
keyword
44379
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"