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Current Treatment Options in Neurology

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01-08-2018 | Neuromuscular Disorders (C Fournier, Section Editor)

Current and Emerging Therapies for Duchenne Muscular Dystrophy

Authors: Megan Crone, MD, FRCPC, Jean K. Mah, MD, MSc, FRCPC

Published in: Current Treatment Options in Neurology | Issue 8/2018

Abstract

Purpose of review

The purpose of this review is to summarize the current and emerging therapies for Duchenne muscular dystrophy (DMD).

Recent findings

Coinciding with new standardized care guidelines, there are a growing number of therapeutic options to treat males with DMD. Treatment of the underlying pathobiology, such as micro-dystrophin gene replacement, exon skipping, stop codon read-through agents, and utrophin modulators showed variable success in animal and human studies. Symptomatic therapies to target muscle ischemia, enhance muscle regeneration, prevent muscle fibrosis, inhibit myostatin, and reduce inflammation are also under investigation.

Summary

DMD is a complex, heterogeneous degenerative disease. The pharmacological and technological achievements made in recent years, plus timely supportive interventions will likely lead to an improved quality of life for many individuals with DMD.

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Title: Current and Emerging Therapies for Duchenne Muscular Dystrophy
Authors: Megan Crone, MD, FRCPC
Jean K. Mah, MD, MSc, FRCPC
Publication date: 01-08-2018
Publisher: Springer US
Published in: Current Treatment Options in Neurology / Issue 8/2018
Print ISSN: 1092-8480
Electronic ISSN: 1534-3138
DOI: https://doi.org/10.1007/s11940-018-0513-6

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Current and Emerging Therapies for Duchenne Muscular Dystrophy

Abstract

Purpose of review

Recent findings

Summary

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Purpose of review

Recent findings

Summary

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