نتایج جستجو برای: deflazacort

تعداد نتایج: 144  

2017
Emanuele Berardi Maurilio Sampaolesi

Muscular dystrophies (MDs) are inherited diseases that affect skeletal and cardiac muscle tissues. Cases range from mild to very severe, resulting in respiratory or cardiac failures. No cures are available for MDs and corticosteroid treatments, mainly deflazacort and prednisolone, only help to control the inflammatory process and slightly delay the progression of the disease. This is due to the...

Journal: :The Journal of pediatrics 2016
Molly M Lamb Nancy A West Lijing Ouyang Michele Yang David Weitzenkamp Katherine James Emma Ciafaloni Shree Pandya Carolyn DiGuiseppi

OBJECTIVES To evaluate growth patterns of ambulatory males with Duchenne muscular dystrophy (DMD) treated with corticosteroids compared with ambulatory, steroid-naïve males with DMD and age-matched unaffected general-population males and to test associations between growth and steroid treatment patterns among treated males. STUDY DESIGN Using data from the Muscular Dystrophy Surveillance, Tra...

Journal: :Human gene therapy 2013
Kevin M Flanigan Katie Campbell Laurence Viollet Wei Wang Ana Maria Gomez Christopher M Walker Jerry R Mendell

Duchenne muscular dystrophy (DMD) typically occurs as a result of truncating mutations in the DMD gene that result in a lack of expression of the dystrophin protein in muscle fibers. Various therapies under development are directed toward restoring dystrophin expression at the subsarcolemmal membrane, including gene transfer. In a trial of intramuscular adeno-associated virus (AAV)-mediated del...

2016
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...

2011
Eun Jung Jang Hyun Jung Jin Young Hee Nam Joo Hee Kim Young-Min Ye Hae-Sim Park

Although corticosteroids have immunosuppressive, anti-inflammatory, and anti-allergic effects, allergic reactions are rare. We report a case involving a 52-year-old-female with acute urticaria caused by oral methylprednisolone. The patient had experienced aspirin-exacerbated respiratory disease (AERD) for 13 years with frequent asthma exacerbations. Symptoms of asthma exacerbations improved wit...

2014
Eun Chae Lee Geun A Kim Ja Wook Koo

Toxic epidermal necrolysis (TEN) is a drug-related fatal disease. Extensive necrosis of the epidermis can lead to serious complications. This report describes two cases of TEN, associated with deflazacort (DFZ), in two boys, aged 4 years and 14 years, with nephrotic syndrome (NS). The 14-year-old male teenager received DFZ following NS relapse. After 17 days, pruritic papules appeared on the lo...

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