Current and Emerging Therapies for Duchenne Muscular Dystrophy

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

(2018) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

A Novel NF-κB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients With Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects

(2017) Joanne M. Donovan et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

Duchenne muscular dystrophy: CRISPR/Cas9 treatment

(2016) Jerry R Mendell et al.   CELL RESEARCH

The importance of genetic diagnosis for Duchenne muscular dystrophy

(2016) Annemieke Aartsma-Rus et al.   JOURNAL OF MEDICAL GENETICS

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy

(2016) Thierry VandenDriessche et al.   MOLECULAR THERAPY

Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

(2016) David Gloss et al.   NEUROLOGY

Histological effects of givinostat in boys with Duchenne muscular dystrophy

(2016) Paolo Bettica et al.   NEUROMUSCULAR DISORDERS

Advances in genetic therapeutic strategies for Duchenne muscular dystrophy

(2015) Simon Guiraud et al.   EXPERIMENTAL PHYSIOLOGY

Combinatorial therapeutic activation with heparin and AICAR stimulates additive effects on utrophin A expression in dystrophic muscles

(2015) Christine Péladeau et al.   HUMAN MOLECULAR GENETICS

Second-generation compound for the modulation of utrophin in the therapy of DMD

(2015) Simon Guiraud et al.   HUMAN MOLECULAR GENETICS

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

Duchenne muscular dystrophy

(2015) Eppie M Yiu et al.   JOURNAL OF PAEDIATRICS AND CHILD HEALTH

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

(2015) Aurélie Goyenvalle et al.   NATURE MEDICINE

Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test

(2015) Marika Pane et al.   NEUROMUSCULAR DISORDERS

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

(2015) Manuel Haas et al.   NEUROMUSCULAR DISORDERS

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

G-CSF supports long-term muscle regeneration in mouse models of muscular dystrophy

(2015) Nozomi Hayashiji et al.   Nature Communications

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

(2015) David G. Ousterout et al.   Nature Communications

Duchenne Muscular Dystrophy

(2014) Marcello Villanova et al.   AMERICAN JOURNAL OF PHYSICAL MEDICINE & REHABILITATION

Halofuginone improves muscle-cell survival in muscular dystrophies

(2014) Anna Bodanovsky et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

A Population-Based Study of Dystrophin Mutations in Canada

(2014) Jean K. Mah et al.   CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy

(2014) Jean K. Mah et al.   NEUROMUSCULAR DISORDERS

Gene Therapy for Muscular Dystrophy: Moving the Field Forward

(2014) Samiah Al-Zaidy et al.   PEDIATRIC NEUROLOGY

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects

(2013) Christopher R. Heier et al.   EMBO Molecular Medicine

Recent progress in satellite cell/myoblast engraftment - relevance for therapy

(2013) Deborah Briggs et al.   FEBS Journal

Reducing CTGF/CCN2 slows down mdx muscle dystrophy and improves cell therapy

(2013) Maria Gabriela Morales et al.   HUMAN MOLECULAR GENETICS

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

The cooperative international neuromuscular research group duchenne natural history study-a longitudinal investigation in the era of glucocorticoid therapy: Design of protocol and the methods used

(2013) Craig M. McDonald et al.   MUSCLE & NERVE

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe

(2013) Erik K. Henricson et al.   MUSCLE & NERVE

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial

(2013) Kevin M. Flanigan et al.   NEUROMUSCULAR DISORDERS

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

(2013) Richard S. Finkel et al.   PLoS One

Functional muscle ischemia in Duchenne and Becker muscular dystrophy

(2013) Gail D. Thomas   Frontiers in Physiology

Attention Deficit Hyperactivity Disorder and Cognitive Function in Duchenne Muscular Dystrophy: Phenotype-Genotype Correlation

(2012) Marika Pane et al.   JOURNAL OF PEDIATRICS

Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophy

(2012) Gunnar M. Buyse et al.   PEDIATRIC PULMONOLOGY

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

Pathophysiology and Therapy of Cardiac Dysfunction in Duchenne Muscular Dystrophy

(2011) Daniel P. Judge et al.   American Journal of Cardiovascular Drugs

Losartan Decreases Cardiac Muscle Fibrosis and Improves Cardiac Function in Dystrophin-Deficient Mdx Mice

(2011) Christopher F. Spurney et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

(2011) Sebahattin Cirak et al.   MOLECULAR THERAPY

Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: Results from a 12 month, double-blind, randomized placebo-controlled trial

(2011) Gunnar M. Buyse et al.   NEUROMUSCULAR DISORDERS

Duchenne Muscular Dystrophy: Continuous Noninvasive Ventilatory Support Prolongs Survival

(2011) J. R. Bach et al.   Respiratory Care

Long-Term Systemic Administration of Unconjugated Morpholino Oligomers for Therapeutic Expression of Dystrophin by Exon Skipping in Skeletal Muscle: Implications for Cardiac Muscle Integrity

(2011) Alberto Malerba et al.   Nucleic Acid Therapeutics

Calcium and the damage pathways in muscular dystrophyThis article is one of a selection of papers published in this special issue on Calcium Signaling.

(2010) David G. Allen et al.   CANADIAN JOURNAL OF PHYSIOLOGY AND PHARMACOLOGY

MicroRNAs Involved in Molecular Circuitries Relevant for the Duchenne Muscular Dystrophy Pathogenesis Are Controlled by the Dystrophin/nNOS Pathway

(2010) Davide Cacchiarelli et al.   Cell Metabolism

Intellect and Behaviour in Duchenne Muscular Dystrophy

(2010) Doris Leibowitz et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

(2010) HaiFang Yin et al.   MOLECULAR THERAPY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

The respiratory management of patients with duchenne muscular dystrophy: A DMD care considerations working group specialty article

(2010) David J. Birnkrant et al.   PEDIATRIC PULMONOLOGY

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

(2010) A. R. Amenta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

(2010) Vinod Malik et al.   Therapeutic Advances in Neurological Disorders

Green tea extract decreases muscle pathology and NF-κB immunostaining in regenerating muscle fibers of mdx mice

(2009) Nicholas P. Evans et al.   CLINICAL NUTRITION

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Mechanisms of Muscle Degeneration, Regeneration, and Repair in the Muscular Dystrophies

(2008) Gregory Q. Wallace et al.   Annual Review of Physiology

Microarray-based mutation detection in thedystrophingene

(2008) Madhuri R. Hegde et al.   HUMAN MUTATION

N-Acetylcysteine ameliorates skeletal muscle pathophysiology inmdxmice

(2008) Nicholas P. Whitehead et al.   JOURNAL OF PHYSIOLOGY-LONDON