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https://www.readbyqxmd.com/read/28987613/treatment-of-cystoid-macular-edema-secondary-to-retinitis-pigmentosa-a-systematic-review
#1
REVIEW
Malini Bakthavatchalam, Frank H P Lai, Shi Song Rong, Danny S Ng, Marten E Brelen
There are various treatments for cystoid macular edema (CME) secondary to retinitis pigmentosa (RP); however, the evidence for these treatments has not been previously systematically reviewed. Our review that includes 23 studies shows that oral carbonic anhydrase inhibitors (CAI) (including acetazolamide, methazolamide) and topical CAI (dorzolamide and brinzolamide) are effective first line treatments. In patients unresponsive to CAI treatment, intravitreal steroids (triamcinolone acetonide and sustained-release dexamethasone implant), oral corticosteroid (Deflazacort), intravitreal anti-vascular endothelial growth factor agents (ranibizumab and bevacizumab), grid laser photocoagulation, pars plana vitrectomy, or ketorolac were also effective in improving CME secondary to RP...
October 4, 2017: Survey of Ophthalmology
https://www.readbyqxmd.com/read/28880848/deflazacort-emflaza-for-duchenne-muscular-dystrophy
#2
(no author information available yet)
No abstract text is available yet for this article.
September 11, 2017: Medical Letter on Drugs and Therapeutics
https://www.readbyqxmd.com/read/28793824/dual-therapy-deflazacort-doxycyclyne-is-better-than-deflazacort-monotherapy-to-alleviate-cardiomyopathy-in-dystrophin-deficient-mdx-mice
#3
Juliano Alves Pereira, Adriana Fogagnolo Mauricio, Maria Julia Marques, Humberto Santo Neto
Cardiomyopathy related to the absence of dystrophin is an important feature in Duchenne muscular dystrophy (DMD) and in the mdx mouse. Doxycycline (DOX) could be a potential therapy for mdx skeletal muscles dystrophy. We investigated whether the corticoid deflazacort (DFZ) plus DOX could improve cardiac mdx dystrophy better than DFZ alone, later (17 months) in dystrophy. Mdx mice (8 months old) received DFZ/DOX or DFZ for 9 months. The combined therapy was greater than DFZ in reducing fibrosis (60% decrease with DFZ/DOX and 40% with DFZ alone) in the right ventricle and transforming growth factor β levels (6...
September 2017: Journal of Cardiovascular Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/28741668/pharmacokinetics-of-deflazacort-in-rabbits-after-intravenous-and-oral-administration-and-its-interaction-with-erythromycin
#4
A I Escudero, P Marín, C M Cárceles, E Escudero
The pharmacokinetic of deflazacort after intravenous and oral administration and the effect of erythromycin on the disposition of deflazacort in rabbits were investigated. A parallel study was carried out in twelve rabbits. The plasma concentration-time profiles of deflazacort were determined after intravenous and oral administration of single dosages of 5 mg/kg in the presence and absence (baseline) of multiple dose erythromycin regimens. Plasma concentrations of 21-desacetyldeflazacort were determined by HPLC...
July 25, 2017: Journal of Veterinary Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/28738778/cardiac-profile-of-asymptomatic-children-with-becker-and-duchenne-muscular-dystrophy-under-treatment-with-steroids-and-with-without-perindopril
#5
Sophie Mavrogeni, Aikaterini Giannakopoulou, Antigoni Papavasiliou, George Markousis-Mavrogenis, Roser Pons, Evangelos Karanasios, Michel Noutsias, Genovefa Kolovou, George Papadopoulos
BACKGROUND: To evaluate cardiovascular function in boys with Duchenne (DMD) and Becker (BMD) muscular dystrophy, using cardiac magnetic resonance (CMR). METHODS: This is a single point cross sectional study of twenty-four boys with genetically ascertained DMD, and 10 with BMD, aged 10.5 ± 1.5 years (range 9-13), were prospectively evaluated by a 1.5 T system and compared with those of age-sex matched controls. The DMD patients were divided in 2 groups. Group A (N = 12) were under treatment with both deflazacort and perindopril, while Group B (n = 12) were under treatment with deflazacort, only...
July 24, 2017: BMC Cardiovascular Disorders
https://www.readbyqxmd.com/read/28690390/integrated-care-of-muscular-dystrophies-in-italy-part-1-pharmacological-treatment-and-rehabilitative-interventions
#6
Luisa Politano, Marianna Scutifero, Melania Patalano, Alessandra Sagliocchi, Antonella Zaccaro, Federica Civati, Erika Brighina, Gianluca Vita, Sonia Messina, Maria Sframeli, Maria Elena Lombardo, Roberta Scalise, Giulia Colia, Maria Catteruccia, Angela Berardinelli, Maria Chiara Motta, Alessandra Gaiani, Claudio Semplicini, Luca Bello, Guja Astrea, Giulia Ricci, Maria Grazia D'Angelo, Giuseppe Vita, Marika Pane, Adele D'Amico, Umberto Balottin, Corrado Angelini, Roberta Battini, Lorenza Magliano
This paper describes the pharmacological therapies and rehabilitative interventions received by 502 patients with Muscular Dystrophies, evaluated in relation to patient's socio-demographic and clinical variables, and geographical areas. Data were collected by the MD-Socio-Demographic and Clinical Schedule (MD-SC-CS) and by the Family Problems Questionnaire (FPQ). The most part of the enrolled patients were in drug treatment. The number of the medications increased in relation to patient's age, disability degree and duration of illness and was higher among patients with Duchenne Muscular Dystrophy (DMD) compared with Becker (BMD) or Limb-Girdle Muscular Dystrophies (LGMD)...
March 2017: Acta Myologica: Myopathies and Cardiomyopathies: Official Journal of the Mediterranean Society of Myology
https://www.readbyqxmd.com/read/28595270/spp1-genotype-and-glucocorticoid-treatment-modify-osteopontin-expression-in-duchenne-muscular-dystrophy-cells
#7
Sara Vianello, Boris Pantic, Aurora Fusto, Luca Bello, Eva Galletta, Doriana Borgia, Bruno F Gavassini, Claudio Semplicini, Gianni Sorarù, Libero Vitiello, Elena Pegoraro
Glucocorticoids are beneficial in Duchenne muscular dystrophy (DMD). Osteopontin (OPN), the protein product of SPP1, plays a role in DMD pathology modulating muscle inflammation and regeneration. A polymorphism in the SPP1 promoter (rs28357094) has been recognized as a genetic modifier of DMD, and there is evidence suggesting that it modifies response to glucocorticoid treatment. The effect of the glucocorticoid deflazacort on SPP1 mRNA and protein expression was investigated in DMD primary human myoblasts and differentiated myotubes with defined rs28357094 genotype (TT versus TG)...
September 1, 2017: Human Molecular Genetics
https://www.readbyqxmd.com/read/28583328/3d-printed-tablets-loaded-with-polymeric-nanocapsules-an-innovative-approach-to-produce-customized-drug-delivery-systems
#8
R C R Beck, P S Chaves, A Goyanes, B Vukosavljevic, A Buanz, M Windbergs, A W Basit, S Gaisford
The generation of multi-functional drug delivery systems, namely solid dosage forms loaded with nano-sized carriers, remains little explored and is still a challenge for formulators. For the first time, the coupling of two important technologies, 3D printing and nanotechnology, to produce innovative solid dosage forms containing drug-loaded nanocapsules was evaluated here. Drug delivery devices were prepared by fused deposition modelling (FDM) from poly(ε-caprolactone) (PCL) and Eudragit(®) RL100 (ERL) filaments with or without a channelling agent (mannitol)...
August 7, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/28574407/muscular-response-to-the-first-three-months-of-deflazacort-treatment-in-boys-with-duchenne-muscular-dystrophy
#9
L Jensen, S J Petersson, N O Illum, H C Laugaard-Jacobsen, T Thelle, L H Jørgensen, H D Schrøder
OBJECTIVE: Duchenne muscular dystrophy (DMD) patients are often treated with glucocorticoids; yet their precise molecular action remains unknown. METHODS: We investigated muscle biopsies from nine boys with DMD (aged: 7,6±2,8 yrs.) collected before and after three months of deflazacort treatment and compared them to eight healthy boys (aged: 5,3±2,4 yrs.). mRNA transcripts involved in activation of satellite cells, myogenesis, regeneration, adipogenesis, muscle growth and tissue inflammation were assessed...
June 1, 2017: Journal of Musculoskeletal & Neuronal Interactions
https://www.readbyqxmd.com/read/28562381/respiratory-involvement-in-neuromuscular-disorders
#10
Matthias Boentert, Stephan Wenninger, Valeria A Sansone
PURPOSE OF REVIEW: In numerous neuromuscular disorders (NMDs), respiratory muscle weakness is present, and acute or chronic respiratory failure may evolve. Very often, respiratory involvement substantially adds to the burden of disease, impairs quality of life, or reduces life expectancy. This article summarizes new aspects of both diagnosis and management of respiratory muscle weakness in patients with NMDs. RECENT FINDINGS: Drugs like deflazacort, ataluren, eteplirsen, and nusinersen are now approved treatments for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy, and others are on their way in NMDs...
October 2017: Current Opinion in Neurology
https://www.readbyqxmd.com/read/28515510/eteplirsen
#11
Danial E Baker
Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are available online to subscribers...
April 2017: Hospital Pharmacy
https://www.readbyqxmd.com/read/28512018/nanoencapsulation-of-a-glucocorticoid-improves-barrier-function-and-anti-inflammatory-effect-on-monolayers-of-pulmonary-epithelial-cell-lines
#12
Lucas A Rigo, Cristiane S Carvalho-Wodarz, Adriana R Pohlmann, Silvia S Guterres, Nicole Schneider-Daum, Claus-Michael Lehr, Ruy C R Beck
The anti-inflammatory effect of polymeric deflazacort nanocapsules (NC-DFZ) was investigated, and possible improvement of epithelial barrier function using filter grown monolayers of Calu-3 cells was assessed. NC prepared from poly(ε-caprolactone) (PCL) had a mean size around 200nm, slightly negative zeta potential (∼-8mV), and low polydispersity index (<0.10). Encapsulation of DFZ had an efficiency of 85%. No cytotoxic effects were observed at particle concentration of 9.85×10(11)NC/ml, which was therefore chosen to evaluate the effect of NC-DFZ at 1% (w/v) of PCL and 0...
October 2017: European Journal of Pharmaceutics and Biopharmaceutics
https://www.readbyqxmd.com/read/28450193/developing-standardized-corticosteroid-treatment-for-duchenne-muscular-dystrophy
#13
Michela Guglieri, Kate Bushby, Michael P McDermott, Kimberly A Hart, Rabi Tawil, William B Martens, Barbara E Herr, Elaine McColl, Jennifer Wilkinson, Janbernd Kirschner, Wendy M King, Michele Eagle, Mary W Brown, Tracey Willis, Deborah Hirtz, Perry B Shieh, Volker Straub, Anne-Marie Childs, Emma Ciafaloni, Russell J Butterfield, Iain Horrocks, Stefan Spinty, Kevin M Flanigan, Nancy L Kuntz, Giovanni Baranello, Helen Roper, Leslie Morrison, Jean K Mah, Adnan Y Manzur, Craig M McDonald, Ulrike Schara, Maja von der Hagen, Richard J Barohn, Craig Campbell, Basil T Darras, Richard S Finkel, Giuseppe Vita, Imelda Hughes, Tiziana Mongini, Elena Pegoraro, Matthew Wicklund, Ekkehard Wilichowski, W Bryan Burnette, James F Howard, Hugh J McMillan, Mathula Thangarajh, Robert C Griggs
Despite corticosteroids being the only treatment documented to improve strength and function in boys with Duchenne muscular dystrophy (DMD) corticosteroid prescription is inconsistent and in some countries, corticosteroids are not prescribed. We are conducting a clinical trial that (1) compares the 3 most frequently prescribed corticosteroid regimes; (2) standardizes treatment of DMD complications; and (3) standardizes prevention of corticosteroid side effects. Investigators at 38 sites in 5 countries plan to recruit 300 boys aged 4-7 who are randomly assigned to one of three regimens: daily prednisone; daily deflazacort; or intermittent prednisone (10days on/10days off)...
July 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28381914/pharmaceutical-approval-update
#14
Mary Choy
Abuse-deterrent hydrocodone bitartrate (Vantrela ER) for long-term opioid treatment; etelcalcetide (Parsabiv) for secondary hyperparathyroidism in patients with chronic kidney disease on hemodialysis; and deflazacort (Emflaza) for Duchenne muscular dystrophy.
April 2017: P & T: a Peer-reviewed Journal for Formulary Management
https://www.readbyqxmd.com/read/28329498/bullous-pemphigoid-of-infancy-report-and-review-of-infantile-and-pediatric-bullous-pemphigoid
#15
REVIEW
Bárbara R Ferreira, Ana S Vaz, Leonor Ramos, José P Reis, Margarida Gonçalo
A 4-month-old infant was observed with an acute itchy bullous dermatosis, predominantly involving the extremities, which revealed a dermal infiltrate rich in eosinophils, C3 deposits at the dermalepidermal junction, and circulating antibodies to BP180 antigen, confirming the diagnosis of bullous pemphigoid. He was initially treated with deflazacort 1 mg/kg/day, further increased to 2 mg/ kg/day, followed by reduction over seven weeks with complete clinical resolution within this period. We discuss epidemiology, etiology, relationship with vaccination, clinical features, and treatment of thisrelatively rare bullous dermatosis in the pediatric age...
February 16, 2017: Dermatology Online Journal
https://www.readbyqxmd.com/read/28274975/deflazacort-approved-for-duchenne-muscular-dystrophy
#16
Kate Traynor
No abstract text is available yet for this article.
March 15, 2017: American Journal of Health-system Pharmacy: AJHP
https://www.readbyqxmd.com/read/28179743/lixisenatide
#17
Danial E Baker, Terri L Levien
Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are are available online to subscribers...
January 2017: Hospital Pharmacy
https://www.readbyqxmd.com/read/28057083/deflazacort-therapeutic-index-relative-potency-and-equivalent-doses-versus-other-corticosteroids
#18
Luca Parente
BACKGROUND: Deflazacort is a synthetic corticosteroid characterized by a favourable pharmacokinetic profile, peculiar pharmacodynamic properties and a good safety profile. However, to the best of our knowledge, no dose-conversion table based on direct comparison of relative potencies between deflazacort and other main corticosteroids is currently available in scientific literature. MAIN BODY: This paper, while reporting a brief review of deflazacort pharmacological properties, its efficacy and tolerability in different clinical areas, has been designed with the specific aim of providing a new dose-conversion table of corticosteroids, including for the first time also deflazacort...
January 5, 2017: BMC Pharmacology & Toxicology
https://www.readbyqxmd.com/read/28043681/long-term-outcome-of-interdisciplinary-management-of-patients-with-duchenne-muscular-dystrophy-receiving-daily-glucocorticoid-treatment
#19
Brenda L Wong, Irina Rybalsky, Karen C Shellenbarger, Cuixia Tian, Mary A McMahon, Meilan M Rutter, Hemant Sawnani, John L Jefferies
OBJECTIVE: To evaluate clinical outcomes and steroid side effects in a cohort of patients with Duchenne muscular dystrophy (DMD) treated with long-term daily glucocorticoid therapy. Although daily glucocorticoid therapy has been shown to extend ambulatory function in DMD, less frequent dosing is often used because of side effect concerns. STUDY DESIGN: Retrospective study of 97 patients with DMD aged 10 to <16 years treated with daily glucocorticoid (89% on deflazacort) for a mean of 8...
March 2017: Journal of Pediatrics
https://www.readbyqxmd.com/read/28018458/successful-treatment-of-tubulointerstitial-nephritis-and-uveitis-with-steroid-and-azathioprine-in-a-12-year-old-boy
#20
Ji Eun Kim, Se Jin Park, Ji Young Oh, Hyeon Joo Jeong, Ji Hong Kim, Jae Il Shin
Tubulointerstitial nephritis and uveitis (TINU) syndrome is a rare disease, often underdiagnosed or misdiagnosed in children. We describe the case of a 12-year-old boy who presented to Severance Hospital with a 1-month history of bilateral conjunctival injection. He was first evaluated by an Ophthalmologist in another hospital and diagnosed with panuveitis. Laboratory tests indicated renal failure, and a renal biopsy confirmed the diagnosis of acute tubulointerstitial nephritis. An extensive exclusion of all possible causes allowed a diagnosis of TINU syndrome...
November 2016: Korean Journal of Pediatrics
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