Duchenne muscular dystrophy: an historical treatment review
Autor(a) principal: | |
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Data de Publicação: | 2019 |
Outros Autores: | , , , , |
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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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 |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2019000800579 |
url |
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2019000800579 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
10.1590/0004-282x20190088 |
dc.rights.driver.fl_str_mv |
info:eu-repo/semantics/openAccess |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
text/html |
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 |
instacron_str |
ABNEURO |
institution |
ABNEURO |
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 |
repository.mail.fl_str_mv |
||revista.arquivos@abneuro.org |
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1754212785477124096 |