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Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

2017; Thieme Medical Publishers (Germany); Volume: 75; Issue: 8 Linguagem: Inglês

10.1590/0004-282x20170112

1678-4227

Abelardo Araújo, Alzira Alves Siqueira Carvalho, Eduardo Boiteux Uchôa Cavalcanti, Jonas Alex Morales Saute, Elmano Carvalho, Marcondes C. França, Alberto Martínez, Monica de M. M. Navarro, Anamarli Nucci, Maria Bernadete Dutra de Resende, Marcus Vinícius Magno Gonçalves, Juliana Gurgel‐Giannetti, Rosana Hermínia Scola, Cláudia Ferreira da Rosa Sobreira, Umbertina Conti Reed, Edmar Zanoteli,

Biotin and Related Studies

Significant advances in the understanding and management of Duchenne muscular dystrophy (DMD) took place since international guidelines were published in 2010. Our objective was to provide an evidence-based national consensus statement for multidisciplinary care of DMD in Brazil. A combination of the Delphi technique with a systematic review of studies from 2010 to 2016 was employed to classify evidence levels and grade of recommendations. Our recommendations were divided in two parts. We present Part 1 here, where we describe the guideline methodology and overall disease concepts, and also provide recommendations on diagnosis, steroid therapy and new drug treatment perspectives for DMD. The main recommendations: 1) genetic testing in diagnostic suspicious cases should be the first line for diagnostic confirmation; 2) patients diagnosed with DMD should have steroids prescribed; 3) lack of published results for phase 3 clinical trials hinders, for now, the recommendation to use exon skipping or read-through agents.