Duchenne muscular dystrophy: an historical treatment review

Detalhes bibliográficos
Autor(a) principal: Werneck,Lineu Cesar
Data de Publicação: 2019
Outros Autores: Lorenzoni,Paulo José, Ducci,Renata Dal-Prá, Fustes,Otto Hernández, Kay,Cláudia Suemi Kamoi, Scola,Rosana Herminia
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Arquivos de neuro-psiquiatria (Online)
Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2019000800579
Resumo: ABSTRACT In this review, we discuss the therapies used in the treatment of patients with Duchenne muscular dystrophy since the first description of the disease. A short description is given of the various theories based on disease pathogenesis, which give the substrates for the many therapeutic interventions. A brief review of the methods of evaluation used in therapeutic trials is made. Of all the treatments, the only drugs that are still considered able to modify the course of the disease are the corticosteroids (prednisone/prednisolone/deflazacort). Other drugs (coenzyme Q10 and creatine) have had a little effect in a few functions without adverse reactions. Idebenone seems to improve the respiratory function in the long term. The trials with mRNA transcription, through nonsense mutations or exon 51 skipping, show some beneficial results in a few functional tests, but they are limited to a small set of DMD patients.
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spelling Duchenne muscular dystrophy: an historical treatment reviewMuscular dystrophyDuchenne dystrophymuscular diseasesABSTRACT In this review, we discuss the therapies used in the treatment of patients with Duchenne muscular dystrophy since the first description of the disease. A short description is given of the various theories based on disease pathogenesis, which give the substrates for the many therapeutic interventions. A brief review of the methods of evaluation used in therapeutic trials is made. Of all the treatments, the only drugs that are still considered able to modify the course of the disease are the corticosteroids (prednisone/prednisolone/deflazacort). Other drugs (coenzyme Q10 and creatine) have had a little effect in a few functions without adverse reactions. Idebenone seems to improve the respiratory function in the long term. The trials with mRNA transcription, through nonsense mutations or exon 51 skipping, show some beneficial results in a few functional tests, but they are limited to a small set of DMD patients.Academia Brasileira de Neurologia - ABNEURO2019-08-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2019000800579Arquivos de Neuro-Psiquiatria v.77 n.8 2019reponame:Arquivos de neuro-psiquiatria (Online)instname:Academia Brasileira de Neurologiainstacron:ABNEURO10.1590/0004-282x20190088info:eu-repo/semantics/openAccessWerneck,Lineu CesarLorenzoni,Paulo JoséDucci,Renata Dal-PráFustes,Otto HernándezKay,Cláudia Suemi KamoiScola,Rosana Herminiaeng2019-09-02T00:00:00Zoai:scielo:S0004-282X2019000800579Revistahttp://www.scielo.br/anphttps://old.scielo.br/oai/scielo-oai.php||revista.arquivos@abneuro.org1678-42270004-282Xopendoar:2019-09-02T00:00Arquivos de neuro-psiquiatria (Online) - Academia Brasileira de Neurologiafalse
dc.title.none.fl_str_mv Duchenne muscular dystrophy: an historical treatment review
title Duchenne muscular dystrophy: an historical treatment review
spellingShingle Duchenne muscular dystrophy: an historical treatment review
Werneck,Lineu Cesar
Muscular dystrophy
Duchenne dystrophy
muscular diseases
title_short Duchenne muscular dystrophy: an historical treatment review
title_full Duchenne muscular dystrophy: an historical treatment review
title_fullStr Duchenne muscular dystrophy: an historical treatment review
title_full_unstemmed Duchenne muscular dystrophy: an historical treatment review
title_sort Duchenne muscular dystrophy: an historical treatment review
author Werneck,Lineu Cesar
author_facet Werneck,Lineu Cesar
Lorenzoni,Paulo José
Ducci,Renata Dal-Prá
Fustes,Otto Hernández
Kay,Cláudia Suemi Kamoi
Scola,Rosana Herminia
author_role author
author2 Lorenzoni,Paulo José
Ducci,Renata Dal-Prá
Fustes,Otto Hernández
Kay,Cláudia Suemi Kamoi
Scola,Rosana Herminia
author2_role author
author
author
author
author
dc.contributor.author.fl_str_mv Werneck,Lineu Cesar
Lorenzoni,Paulo José
Ducci,Renata Dal-Prá
Fustes,Otto Hernández
Kay,Cláudia Suemi Kamoi
Scola,Rosana Herminia
dc.subject.por.fl_str_mv Muscular dystrophy
Duchenne dystrophy
muscular diseases
topic Muscular dystrophy
Duchenne dystrophy
muscular diseases
description ABSTRACT In this review, we discuss the therapies used in the treatment of patients with Duchenne muscular dystrophy since the first description of the disease. A short description is given of the various theories based on disease pathogenesis, which give the substrates for the many therapeutic interventions. A brief review of the methods of evaluation used in therapeutic trials is made. Of all the treatments, the only drugs that are still considered able to modify the course of the disease are the corticosteroids (prednisone/prednisolone/deflazacort). Other drugs (coenzyme Q10 and creatine) have had a little effect in a few functions without adverse reactions. Idebenone seems to improve the respiratory function in the long term. The trials with mRNA transcription, through nonsense mutations or exon 51 skipping, show some beneficial results in a few functional tests, but they are limited to a small set of DMD patients.
publishDate 2019
dc.date.none.fl_str_mv 2019-08-01
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
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dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 10.1590/0004-282x20190088
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dc.publisher.none.fl_str_mv Academia Brasileira de Neurologia - ABNEURO
publisher.none.fl_str_mv Academia Brasileira de Neurologia - ABNEURO
dc.source.none.fl_str_mv Arquivos de Neuro-Psiquiatria v.77 n.8 2019
reponame:Arquivos de neuro-psiquiatria (Online)
instname:Academia Brasileira de Neurologia
instacron:ABNEURO
instname_str Academia Brasileira de Neurologia
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reponame_str Arquivos de neuro-psiquiatria (Online)
collection Arquivos de neuro-psiquiatria (Online)
repository.name.fl_str_mv Arquivos de neuro-psiquiatria (Online) - Academia Brasileira de Neurologia
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