دورية أكاديمية

Articles Treatment of Duchenne muscular dystrophy with

التفاصيل البيبلوغرافية
العنوان: Articles Treatment of Duchenne muscular dystrophy with
المؤلفون: Janbernd Kirschner, Joachim Schessl, Ulrike Schara, Bernd Reitter, Georg M Stettner, Elke Hobbiebrunken, Ekkehard Wilichowski, Jürg Lütschg, Claudia Schmoor, Gabriele Ihorst, Rudolf Korinthenberg
المساهمون: The Pennsylvania State University CiteSeerX Archives
المصدر: http://www.distrofiamuscular.net/ciclosporin_trial.PDFTest.
سنة النشر: 2010
المجموعة: CiteSeerX
الوصف: Background Duchenne muscular dystrophy is a rare X-linked progressive disease characterised by loss of ambulation at about age 10 years, with death in early adulthood due to respiratory and cardiac insuffi ciency. Steroids are eff ective at slowing the progression of muscle weakness; however, their use is limited by side-eff ects, prompting the search for alternatives. We assessed the eff ect of ciclosporin A as monotherapy and in combination with intermittent prednisone for the treatment of ambulant patients with this disorder. Methods Our study was a parallel-group, placebo-controlled, double-blind, multicentre trial at trial sites of the German muscular dystrophy network, MD-NET, over 36 months. Ambulant patients with Duchenne muscular dystrophy who were aged 5 years or older were randomly assigned to receive either ciclosporin A (3·5–4·0 mg/kg per day) or matching placebo. Allocation was done centrally with computer-generated random numbers. Patients and investigators were masked to the allocated treatment. After 3 months of treatment, both groups were also given intermittent prednisone for a further 12 months (0·75 mg/kg, alternating 10 days on with 10 days off). All patients who received at least one dose of study drug or placebo were included in the primary analysis. The primary outcome measure was manual muscle strength measured on the Medical Research Council (MRC) scale. This trial is registered with the German clinical trial register DRKS, number DRKS00000445. Findings 77 patients were randomly assigned to the ciclosporin A group and 76 to the placebo group; 73 patients on
نوع الوثيقة: text
اللغة: English
العلاقة: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.620.54Test; http://www.distrofiamuscular.net/ciclosporin_trial.PDFTest
الإتاحة: http://www.distrofiamuscular.net/ciclosporin_trial.PDFTest
حقوق: Metadata may be used without restrictions as long as the oai identifier remains attached to it.
رقم الانضمام: edsbas.44492F9C
قاعدة البيانات: BASE