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Drugs in development and dietary approach for Duchenne muscular dystrophy

Authors Angelini C, Tasca E

Received 11 April 2015

Accepted for publication 11 May 2015

Published 11 August 2015 Volume 2015:5 Pages 51—60


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Editor who approved publication: Dr Lise Aagaard

Corrado Angelini, Elisabetta Tasca

Neuromuscular Laboratory, Fondazione San Camillo Hospital IRCCS, Venice, Italy

Abstract: Therapeutic trials studying Duchenne muscular dystrophy (DMD) in Europe and the USA have been done using a protocol that includes manual muscle testing and functional testing, and have shown the efficacy of steroid drugs in various doses and regimens. Further, drisapersen and eteplirsen (exon skipping drugs) and ataluren (a drug to overcome stop codon mutations) have achieved some clinical improvement. Cardioprotective drugs are efficacious in DMD, and eplerenone, an aldosterone inhibitor and diuretic, is now being used to treat the disease. The dietary approach should be used in wheelchair-bound DMD children in combination with respiratory assistance. The importance of some of the treatments proposed is that they might also be useful in other genetic disorders where stop codon mutations are present; moreover, it is possible that these new treatments will improve quality of life for many patients.

Keywords: Duchenne muscular dystrophy, steroids, ataluren, drisapersen, eplerenone, eteplirsen

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