keyword
MENU ▼
Read by QxMD icon Read
search

MPS 1-H/S

keyword
https://www.readbyqxmd.com/read/27761411/30-months-follow-up-of-an-early-enzyme-replacement-therapy-in-a-severe-morquio-a-patient-about-one-case
#1
J Do Cao, A Wiedemann, T Quinaux, S F Battaglia-Hsu, L Mainard, R Froissart, C Bonnemains, S Ragot, B Leheup, P Journeau, F Feillet
: Patients under 5 years were not evaluated in the phase-3 study for enzyme replacement therapy (ERT) in MPS IV A. Here we describe the evolution of a severe Morquio A pediatric patient who was diagnosed at 19 months old and treated by ERT at 21 months old for the next 30 months. Applying the standard ERT protocol on this very young patient appeared to reduce his urinary excretion of glycosaminoglycans (GAGs); the improvements in both the 6 minute-walk test (6MWT) and the stair climb test, however, were no different than those reported in the nature history study...
December 2016: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/27733086/comparative-study-of-kanamycin-sulphate-microparticles-and-nanoparticles-for-intramuscular-administration-preparation-in-vitro-release-and-preliminary-in-vivo-evaluation
#2
Sanaul Mustafa, V Kusum Devi, Roopa S Pai
Kanamycin sulphate (KS) is a Mycobacterium tuberculosis protein synthesis inhibitor. KS is polycationic, a property responsible for KS poor oral absorption half-life (2.5 h) and rapid renal clearance, which results in serious nephrotoxicity/ototoxicity. The current study aimed to develop KS-loaded PLGA vitamin-E-TPGS microparticles (MPs) and nanoparticles (NPs) to reduce the dosing frequency and dose-related adverse effect. In vitro release was sustained up to 10 days for KS PLGA-TPGS MPs and 13 days for KS PLGA-TPGS NPs in phosphate-buffered saline (PBS) pH 7...
November 1, 2016: Journal of Microencapsulation
https://www.readbyqxmd.com/read/27650774/enhanced-procoagulant-activity-on-blood-cells-after-acute-ischemic-stroke
#3
Zhipeng Yao, Lixiu Wang, Xiaoming Wu, Lu Zhao, Chunling Chi, Li Guo, Dongxia Tong, Xiaoyan Yang, Zengxiang Dong, Ruijuan Deng, Valerie A Novakovic, Hemant S Thatte, Yayan Bi, Ye Tian, Jialan Shi, Jin Zhou, Junjie Kou, Shaoshan Hu
The role of phosphatidylserine (PS)-mediated procoagulant activity (PCA) in stroke remains unclear. To ascertain this role, early dynamic evolution of PS exposure on blood cells and released microparticles (MPs) and the corresponding PCA were evaluated in patients with acute ischemic stroke (AIS). Flow cytometry analyses revealed that initial levels of PS exposure on erythrocyte, platelet, and leukocyte were 2.40-, 1.36-, and 1.38-fold higher, respectively, in AIS than the risk factor-matched (RF) controls...
September 20, 2016: Translational Stroke Research
https://www.readbyqxmd.com/read/27590925/a-prospective-one-year-natural-history-study-of-mucopolysaccharidosis-types-iiia-and-iiib-implications-for-clinical-trial-design
#4
K V Truxal, H Fu, D M McCarty, K A McNally, K L Kunkler, N A Zumberge, L Martin, S C Aylward, L N Alfano, K M Berry, L P Lowes, M Corridore, C McKee, K L McBride, K M Flanigan
Mucopolysaccharidosis type III is a group of four autosomal recessive enzyme deficiencies leading to tissue accumulation of heparan sulfate. Central nervous system disease is prominent, with initial normal development followed by neurocognitive decline leading to death. In order to define outcome measures suitable for gene transfer trials, we prospectively assessed disease progression in MPS IIIA and IIIB subjects >2years old at three time points over one year (baseline, 6 and 12months). Fifteen IIIA (9 male, 6 female; age 5...
November 2016: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/27569936/pilot-randomized-trial-of-tacrolimus-everolimus-vs-tacrolimus-enteric-coated-mycophenolate-sodium-in-adult-primary-kidney-transplant-recipients-at-a-single-center
#5
G Ciancio, P Tryphonopoulos, J J Gaynor, G Guerra, J Sageshima, D Roth, L Chen, W Kupin, A Mattiazzi, L Tueros, S Flores, L Hanson, R H Powell, P Ruiz, R Vianna, G W Burke
BACKGROUND: Recent studies suggest that the combination of tacrolimus (TAC) and everolimus (EVL) could become a viable option for use as standard maintenance immunosuppression in non-highly sensitized kidney transplant recipients. METHODS: We conducted a single-center, open-label, randomized pilot trial comparing two maintenance immunosuppression regimens in non-highly sensitized, adult, primary kidney transplant recipients: (TAC/EVL, Group A) vs our standard maintenance regimen of TAC plus enteric-coated mycophenolate mofetil (TAC/EC-MPS, Group B)...
July 2016: Transplantation Proceedings
https://www.readbyqxmd.com/read/27511985/the-response-of-muscle-protein-synthesis-following-whole-body-resistance-exercise-is-greater-following-40%C3%A2-g-than-20%C3%A2-g-of-ingested-whey-protein
#6
Lindsay S Macnaughton, Sophie L Wardle, Oliver C Witard, Chris McGlory, D Lee Hamilton, Stewart Jeromson, Clare E Lawrence, Gareth A Wallis, Kevin D Tipton
The currently accepted amount of protein required to achieve maximal stimulation of myofibrillar protein synthesis (MPS) following resistance exercise is 20-25 g. However, the influence of lean body mass (LBM) on the response of MPS to protein ingestion is unclear. Our aim was to assess the influence of LBM, both total and the amount activated during exercise, on the maximal response of MPS to ingestion of 20 or 40 g of whey protein following a bout of whole-body resistance exercise. Resistance-trained males were assigned to a group with lower LBM (≤65 kg; LLBM n = 15) or higher LBM (≥70 kg; HLBM n = 15) and participated in two trials in random order...
August 2016: Physiological Reports
https://www.readbyqxmd.com/read/27198001/the-efficacy-and-safety-of-intensified-enteric-coated-mycophenolate-sodium-with-low-exposure-of-calcineurin-inhibitors-in-chinese-de-novo-kidney-transplant-recipients-a-prospective-study
#7
H Zhang, L Liu, J Li, Q Fu, J Wan, R Deng, H Wang, J Liao, W Deng, S Deng, L Chen, C Wang
AIMS: The aim of this study was to investigate the efficacy and safety of a transient intensified enteric-coated mycophenolate sodium (EC-MPS) dosing regimen with low exposure of calcineurin inhibitors (CNIs) in Chinese de novo kidney transplantation. METHODS: In a 6-month prospective study, a total of 97 recipients were enrolled and assigned to either an intensified EC-MPS dosing (IS) regimen or a standard EC-MPS dosing (SD) regimen. The area under the curve (AUC) of MPA was assessed at week 1 post transplant...
June 2016: International Journal of Clinical Practice
https://www.readbyqxmd.com/read/27161681/oc-09-microparticle-mediated-transfer-of-tf-hypercoagulability-between-cancer-cells
#8
H Shaker, A S Rothmeier, C C Kirwan, W Ruf
INTRODUCTION: Tissue Factor (TF) promotes apoptosis-resistance and facilitates haematogenous metastasis. Cancer cells release TF-bearing microparticles (TF-MP) which have pro-coagulant activity. TF-MPs from cancer cells induce increase in TF expression and procoagulant activity of recipient endothelial cells. Drug resistance microparticles transfer between cancer cells, resulting in induction of recipient cell drug resistance, however transfer of TF-MPs between cancer cells has not been demonstrated...
April 2016: Thrombosis Research
https://www.readbyqxmd.com/read/26908836/clinical-course-of-sly-syndrome-mucopolysaccharidosis-type-vii
#9
Adriana M Montaño, Ngu Lock-Hock, Robert D Steiner, Brett H Graham, Marina Szlago, Robert Greenstein, Mercedes Pineda, Antonio Gonzalez-Meneses, Mahmut Çoker, Dennis Bartholomew, Mark S Sands, Raymond Wang, Roberto Giugliani, Alfons Macaya, Gregory Pastores, Anastasia K Ketko, Fatih Ezgü, Akemi Tanaka, Laila Arash, Michael Beck, Rena E Falk, Kaustuv Bhattacharya, José Franco, Klane K White, Grant A Mitchell, Loreta Cimbalistiene, Max Holtz, William S Sly
BACKGROUND: Mucopolysaccharidosis VII (MPS VII) is an ultra-rare disease characterised by the deficiency of β-glucuronidase (GUS). Patients' phenotypes vary from severe forms with hydrops fetalis, skeletal dysplasia and mental retardation to milder forms with fewer manifestations and mild skeletal abnormalities. Accurate assessments on the frequency and clinical characteristics of the disease have been scarce. The aim of this study was to collect such data. METHODS: We have conducted a survey of physicians to document the medical history of patients with MPS VII...
June 2016: Journal of Medical Genetics
https://www.readbyqxmd.com/read/26634710/risk-factors-for-contact-lens-related-microbial-keratitis-in-singapore
#10
C H L Lim, N A Carnt, M Farook, J Lam, D T Tan, J S Mehta, F Stapleton
PURPOSE: To investigate independent risk factors for contact lens-related microbial keratitis in Singapore and estimate their impact on disease load. METHODS: Cases were contact lens wearers presenting to Singapore National Eye Centre with microbial keratitis between 2008 and 2010. Community contact lens wearers were recruited as controls. All wearers completed a previously validated questionnaire describing contact lens wear history, hygiene and compliance habits, and demographics...
March 2016: Eye
https://www.readbyqxmd.com/read/26529320/methylprednisolone-for-the-treatment-of-patients-with-acute-spinal-cord-injuries-a-systematic-review-and-meta-analysis
#11
Nathan Evaniew, Emilie P Belley-Côté, Nader Fallah, Vanessa K Noonan, Carly S Rivers, Marcel F Dvorak
Previous meta-analyses of methylprednisolone (MPS) for patients with acute traumatic spinal cord injuries (TSCIs) have not addressed confidence in the quality of evidence used for pooled effect estimates, and new primary studies have been recently published. We aimed to determine whether MPS improves motor recovery and is associated with increased risks for adverse events. We searched MEDLINE, EMBASE, and The Cochrane Library, and two reviewers independently screened articles, extracted data, and evaluated risk of bias...
March 1, 2016: Journal of Neurotrauma
https://www.readbyqxmd.com/read/26496097/thrombin-generation-and-procoagulant-microparticle-profiles-after-acute-trauma-a-prospective-cohort-study
#12
COMPARATIVE STUDY
Myung S Park, Ailing Xue, Grant M Spears, Timothy M Halling, Michael J Ferrara, Melissa M Kuntz, Sabtir K Dhillon, Donald H Jenkins, William S Harmsen, Karla V Ballman, Paul Harrison, John A Heit
OBJECTIVE: The two sides of trauma-induced coagulopathy, the hypocoagulable and the hypercoagulable states, are poorly understood. To identify potential mechanisms for venous thromboembolism and bleeding after acute trauma, we estimated changes in circulating procoagulant microparticles (MPs) and thrombin activity during hospitalization for trauma. METHODS: Whole blood was collected by venipuncture into 3.2% trisodium citrate at 0, 6, 12, 24, and 72 hours after injury and discharge...
November 2015: Journal of Trauma and Acute Care Surgery
https://www.readbyqxmd.com/read/26488552/inhibition-of-heme-oxygenase-activity-using-a-microparticle-formulation-of-zinc-protoporphyrin-in-an-acute-hemolytic-newborn-mouse-model
#13
Kazumichi Fujioka, Flora Kalish, Ronald J Wong, David K Stevenson
BACKGROUND: Increased bilirubin production due to hemolysis can lead to neonatal hyperbilirubinemia. Inhibition of heme oxygenase (HO), the rate-limiting enzyme in heme catabolism, by metalloporphyrins (Mps) may be an ideal preventive strategy for neonatal hemolytic disease. Zinc protoporphyrin (ZnPP) is a naturally occurring Mp, potent, not phototoxic, with minimal HO-1 upregulation, but is not orally absorbed. Recently, we designed a lipid-based ZnPP formulation (ZnPP-Lipid), which is orally absorbed by newborn mice...
February 2016: Pediatric Research
https://www.readbyqxmd.com/read/26279095/a-combination-hydrogel-microparticle-based-vaccine-prevents-type-1-diabetes-in-non-obese-diabetic-mice
#14
Young Mee Yoon, Jamal S Lewis, Matthew R Carstens, Martha Campbell-Thompson, Clive H Wasserfall, Mark A Atkinson, Benjamin G Keselowsky
Targeted delivery of self-antigens to the immune system in a mode that stimulates a tolerance-inducing pathway has proven difficult. To address this hurdle, we developed a vaccine based-approach comprised of two synthetic controlled-release biomaterials, poly(lactide-co-glycolide; PLGA) microparticles (MPs) encapsulating denatured insulin (key self-antigen in type 1 diabetes; T1D), and PuraMatrix(TM) peptide hydrogel containing granulocyte macrophage colony-stimulating factor (GM-CSF) and CpG ODN1826 (CpG), which were included as vaccine adjuvants to recruit and activate immune cells...
2015: Scientific Reports
https://www.readbyqxmd.com/read/26052536/a-novel-long-lived-and-highly-engraftable-immunodeficient-mouse-model-of-mucopolysaccharidosis-type-i
#15
Daniel C Mendez, Alexander E Stover, Anthony D Rangel, David J Brick, Hubert E Nethercott, Marissa A Torres, Omar Khalid, Andrew Ms Wong, Jonathan D Cooper, James V Jester, Edwin S Monuki, Cian McGuire, Steven Q Le, Shih-Hsin Kan, Patricia I Dickson, Philip H Schwartz
Mucopolysaccharidosis type I (MPS I) is an inherited α-L-iduronidase (IDUA, I) deficiency in which glycosaminoglycan (GAG) accumulation causes progressive multisystem organ dysfunction, neurological impairment, and death. Current MPS I mouse models, based on a NOD/SCID (NS) background, are short-lived, providing a very narrow window to assess the long-term efficacy of therapeutic interventions. They also develop thymic lymphomas, making the assessment of potential tumorigenicity of human stem cell transplantation problematic...
2015: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/25842187/a-combination-dual-sized-microparticle-system-modulates-dendritic-cells-and-prevents-type-1-diabetes-in-prediabetic-nod-mice
#16
Jamal S Lewis, Natalia V Dolgova, Ying Zhang, Chang Qing Xia, Clive H Wasserfall, Mark A Atkinson, Michael J Clare-Salzler, Benjamin G Keselowsky
We developed a novel poly(lactic-co-glycolic acid)-based, microparticle (MP) system providing concurrent delivery of multiple encapsulated immuno-suppressive factors and antigen, for in vivo conditioning of dendritic cells (DCs) toward a tolerance promoting pathway. Subcutaneous administration prevents onset of type 1 diabetes (T1D) in NOD mice. Two MP sizes were made: phagocytosable MPs were fabricated encapsulating vitamin D3 or insulin B(9-23) peptide, while unphagocytosable MPs were fabricated encapsulating TGF-β1 or GM-CSF...
September 2015: Clinical Immunology: the Official Journal of the Clinical Immunology Society
https://www.readbyqxmd.com/read/25827594/intake-of-low-dose-leucine-rich-essential-amino-acids-stimulates-muscle-anabolism-equivalently-to-bolus-whey-protein-in-older-women-at-rest-and-after-exercise
#17
RANDOMIZED CONTROLLED TRIAL
Syed S I Bukhari, Bethan E Phillips, Daniel J Wilkinson, Marie C Limb, Debbie Rankin, William K Mitchell, Hisamine Kobayashi, Paul L Greenhaff, Kenneth Smith, Philip J Atherton
Dysregulated anabolic responses to nutrition/exercise may contribute to sarcopenia; however, these characteristics are poorly defined in female populations. We determined the effects of two feeding regimes in older women (66 ± 2.5 yr; n = 8/group): bolus whey protein (WP-20 g) or novel low-dose leucine-enriched essential amino acids (EAA) [LEAA; 3 g (40% leucine)]. Using [(13)C6]phenylalanine infusions, we quantified muscle (MPS) and albumin (APS) protein synthesis at baseline and in response to both feeding (FED) and feeding plus exercise (FED-EX; 6 × 8 knee extensions at 75% 1-repetition maximum)...
June 15, 2015: American Journal of Physiology. Endocrinology and Metabolism
https://www.readbyqxmd.com/read/25705884/caspase-1-mediated-pathway-promotes-generation-of-thromboinflammatory-microparticles
#18
Andrea S Rothmeier, Patrizia Marchese, Brian G Petrich, Christian Furlan-Freguia, Mark H Ginsberg, Zaverio M Ruggeri, Wolfram Ruf
Extracellular ATP is a signal of tissue damage and induces macrophage responses that amplify inflammation and coagulation. Here we demonstrate that ATP signaling through macrophage P2X7 receptors uncouples the thioredoxin (TRX)/TRX reductase (TRXR) system and activates the inflammasome through endosome-generated ROS. TRXR and inflammasome activity promoted filopodia formation, cellular release of reduced TRX, and generation of extracellular thiol pathway-dependent, procoagulant microparticles (MPs). Additionally, inflammasome-induced activation of an intracellular caspase-1/calpain cysteine protease cascade degraded filamin, thereby severing bonds between the cytoskeleton and tissue factor (TF), the cell surface receptor responsible for coagulation activation...
April 2015: Journal of Clinical Investigation
https://www.readbyqxmd.com/read/25604073/oxidation-initiated-myosin-subfragment-cross-linking-and-structural-instability-differences-between-white-and-red-muscle-fiber-types
#19
COMPARATIVE STUDY
Changqi Liu, Youling L Xiong
Both white and red muscles are commonly used in meat processing, and protein cross-linking, which may be affected by oxidants, is a key factor affecting the product quality. In this study, myofibrillar proteins (MPs) extracted from postrigor chicken Pectoralis major (PM, predominantly white) and Gastrocnemius (GN, predominantly red) muscles were subjected to a •OH-oxidizing system (10 μM FeCl3 , 0.1 mM ascorbic acid, with 0, 5, 10, or 20 mM H2 O2 ) at pH 6.2, 4 °C for 18 h. The solubility of nonoxidized (control) PM MPs (63%) was higher than that of control GN MPs (41%)...
February 2015: Journal of Food Science
https://www.readbyqxmd.com/read/25494416/transfusion-of-stored-red-blood-cells-in-trauma-patients-is-not-associated-with-increased-procoagulant-microparticles
#20
Satbir K Dhillon, Mindy L Houck, Donald H Jenkins, Jordan K Rosedahl, William S Harmsen, Timothy M Halling, Myung S Park
BACKGROUND: We set out to determine the effects of transfusing stored red blood cells (RBCs) on the levels of procoagulant microparticles (MPs) in the blood of trauma patients. METHODS: Blood was drawn and processed to platelet poor plasma for MP analysis for 409 injured patients seen in the trauma bay from February 2011 to January 2013. Blood from 27 noninjured volunteers was also analyzed. Quantification of total procoagulant MP (per microliter plasma) using a direct plasma analysis via flow cytometry was performed...
November 2014: Journal of Trauma and Acute Care Surgery
keyword
keyword
106257
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"