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https://www.readbyqxmd.com/read/28690390/integrated-care-of-muscular-dystrophies-in-italy-part-1-pharmacological-treatment-and-rehabilitative-interventions
#1
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
#2
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)...
June 8, 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
#3
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)...
June 3, 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
#4
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
#5
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...
May 29, 2017: Current Opinion in Neurology
https://www.readbyqxmd.com/read/28515510/eteplirsen
#6
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
#7
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 A549 and Calu-3 using as models was assessed. NC prepared from poly(ε-caprolactone) (PCL) had a mean size around 200 nm, slightly negative zeta potential (∼ - 8 mV), 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 x 10(11) NC/ml, which was therefore chosen to evaluate the effect of NC-DFZ at 1% (w/v) of PCL and 0...
May 13, 2017: European Journal of Pharmaceutics and Biopharmaceutics
https://www.readbyqxmd.com/read/28450193/developing-standardized-corticosteroid-treatment-for-duchenne-muscular-dystrophy
#8
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
#9
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
#10
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
#11
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
#12
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
#13
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
#14
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
#15
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
https://www.readbyqxmd.com/read/28018451/eosinophilic-gastroenteritis-in-an-18-year-old-male-with-prolonged-nephrotic-syndrome
#16
Da Min Choi, Jung Eun Pyun, Hyung Eun Yim, Kee Hwan Yoo, Jung Ok Shim, Eun Jung Lee, Nam Hee Won
Eosinophilic gastroenteritis is a rare disease characterized by prominent eosinophilic tissue infiltration of the gastrointestinal tract. Here, we report a case of eosinophilic gastroenteritis in an 18-year-old patient with prolonged nephrotic syndrome who presented with abdominal pain and peripheral hypereosinophilia. During the previous 2 years, he had visited local Emergency Department several times because of epigastric pain and nausea. He had been treated with steroid-dependent nephrotic syndrome since 3 years of age...
November 2016: Korean Journal of Pediatrics
https://www.readbyqxmd.com/read/27843100/isolation-and-structural-characterization-of-novel-photolytic-degradation-impurities-of-deflazacort-using-q-tof-2d-nmr-and-ftir
#17
Ch Krishnam Raju, Avadhesh Kumar Pandey, Kaushik Ghosh, Arunima Pola, Sanath Kumar Goud, Mrunal A Jaywant, Sameer G Navalgund, Koduru V Surendranath
Forced Degradation of Deflazacort drug substance in ultraviolet light condition resulted into a number of significant degradation products. Two of these degradation products were found to be unknown during the study and marked as DD-I and DD-II. Thus, the objective of this work is to investigate and identify these two novel degradation products of DFZ. The isolation method for these new degradation products were developed using a new reverse-phase high performance liquid chromatography (HPLC). DD-I and DD-II, eluting at 0...
January 30, 2017: Journal of Pharmaceutical and Biomedical Analysis
https://www.readbyqxmd.com/read/27746500/retrospective-cohort-study-comparing-the-efficacy-of-prednisolone-and-deflazacort-in-children-with-muscular-dystrophy-a-6-years-experience-in-a-south-indian-teaching-hospital
#18
Harish Petnikota, Vrisha Madhuri, Sangeet Gangadharan, Indira Agarwal, Belavendra Antonisamy
BACKGROUND: Muscular dystrophies are inherited myogenic disorders characterized by progressive muscle wasting and weakness of variable distribution and severity. They are a heterogeneous group characterized by variable degree of skeletal and cardiac muscle involvement. The most common and the most severe form of muscular dystrophy is DMD. Currently, there is no curative treatment for muscular dystrophies. Several drugs have been studied to retard the progression of the muscle weakness...
September 2016: Indian Journal of Orthopaedics
https://www.readbyqxmd.com/read/27566742/efficacy-and-safety-of-deflazacort-vs-prednisone-and-placebo-for-duchenne-muscular-dystrophy
#19
RANDOMIZED CONTROLLED TRIAL
Robert C Griggs, J Phillip Miller, Cheryl R Greenberg, Darcy L Fehlings, Alan Pestronk, Jerry R Mendell, Richard T Moxley, Wendy King, John T Kissel, Valerie Cwik, Michel Vanasse, Julaine M Florence, Shree Pandya, Jordan S Dubow, James M Meyer
OBJECTIVE: To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD). METHODS: This phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5-15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups...
November 15, 2016: Neurology
https://www.readbyqxmd.com/read/27542440/identifying-evidence-of-cardio-renal-syndrome-in-patients-with-duchenne-muscular-dystrophy-using-cystatin-c
#20
Chet R Villa, Ahmad Kaddourah, Jacob Mathew, Thomas D Ryan, Brenda L Wong, Stuart L Goldstein, John L Jefferies
Patients with Duchenne muscular dystrophy (DMD) develop dilated cardiomyopathy and are at risk for kidney injury. Creatinine based estimated glomerular filtration rate (eGFR) is limited by low muscle mass with low serum creatinine levels in DMD. We assessed the relationship between cardiac function, modified Schwartz eGFR and cystatin C eGFR in patients with DMD. Ninety-three patients with DMD were screened for renal dysfunction in an outpatient neuromuscular clinic. Patients with new nephrotoxic medications, recent hospitalization or decompensated heart failure were excluded from the analysis...
October 2016: Neuromuscular Disorders: NMD
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