Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy

Identification of Disease Specific Pathways Usingin VivoSILAC Proteomics in Dystrophin DeficientmdxMouse

(2013) Sree Rayavarapu et al.   MOLECULAR & CELLULAR PROTEOMICS

24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy

(2013) Elena Stacy Mazzone et al.   PLoS One

Pharmacologically Targeting the Primary Defect and Downstream Pathology in Duchenne Muscular Dystrophy

(2012) Rebecca J. Fairclough et al.   CURRENT GENE THERAPY

A critical review of functional assessment tools for upper limbs in Duchenne muscular dystrophy

(2012) ELENA S MAZZONE et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

microRNA-206 promotes skeletal muscle regeneration and delays progression of Duchenne muscular dystrophy in mice

(2012) Ning Liu et al.   JOURNAL OF CLINICAL INVESTIGATION

Nitric Oxide in Myogenesis and Therapeutic Muscle Repair

(2012) Clara De Palma et al.   MOLECULAR NEUROBIOLOGY

Loss of miR-29 in Myoblasts Contributes to Dystrophic Muscle Pathogenesis

(2012) Lijun Wang et al.   MOLECULAR THERAPY

Upregulation of chemokines and their receptors in duchenne muscular dystrophy: potential for attenuation of myofiber necrosis

(2012) Boel De Paepe et al.   MUSCLE & NERVE

Nitric oxide donor and non steroidal anti inflammatory drugs as a therapy for muscular dystrophies: Evidence from a safety study with pilot efficacy measures in adult dystrophic patients

(2012) Maria Grazia D’Angelo et al.   PHARMACOLOGICAL RESEARCH

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

(2012) Y. Aoki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy

(2012) G. C. Kendall et al.   Science Translational Medicine

Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy

(2012) E. A. Martin et al.   Science Translational Medicine

Exon Skipping Quantification by Quantitative Reverse-Transcription Polymerase Chain Reaction in Duchenne Muscular Dystrophy Patients Treated with the Antisense Oligomer Eteplirsen

(2012) Karen Anthony et al.   Human Gene Therapy Methods

Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy

(2012) Thomas C Roberts 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

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

(2011) K. Anthony et al.   BRAIN

Impending therapies for Duchenne muscular dystrophy

(2011) Terence A. Partridge   CURRENT OPINION IN NEUROLOGY

Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy

(2011) ANNA MAYHEW et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

miRNAs as serum biomarkers for Duchenne muscular dystrophy

(2011) Davide Cacchiarelli et al.   EMBO Molecular Medicine

Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up

(2011) Francesca Magri et al.   JOURNAL OF NEUROLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

(2011) Christophe Pichavant et al.   MOLECULAR THERAPY

Cardiomyopathy of duchenne muscular dystrophy: Current understanding and future directions

(2011) Christopher F. Spurney   MUSCLE & NERVE

Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study

(2011) E. Mazzone et al.   NEUROLOGY

Muscle histology vs MRI in Duchenne muscular dystrophy

(2011) M. Kinali et al.   NEUROLOGY

P4.10 The relationship of ataluren plasma concentration and response across clinical studies in nonsense mutation dystrophinopathy

(2011) R. Finkel et al.   NEUROMUSCULAR DISORDERS

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

Multiple exon skipping strategies to by-pass dystrophin mutations

(2011) Carl F. Adkin et al.   NEUROMUSCULAR DISORDERS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

A dual acting compound releasing nitric oxide (NO) and ibuprofen, NCX 320, shows significant therapeutic effects in a mouse model of muscular dystrophy

(2011) Clara Sciorati et al.   PHARMACOLOGICAL RESEARCH

Identification of Muscle-Specific MicroRNAs in Serum of Muscular Dystrophy Animal Models: Promising Novel Blood-Based Markers for Muscular Dystrophy

(2011) Hideya Mizuno et al.   PLoS One

iNOS Ablation Does Not Improve Specific Force of the Extensor Digitorum Longus Muscle in Dystrophin-Deficient mdx4cv Mice

(2011) Dejia Li et al.   PLoS One

Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy

(2011) F. S. Tedesco et al.   Science Translational Medicine

Nitric Oxide Sustains Long-Term Skeletal Muscle Regeneration by Regulating Fate of Satellite Cells Via Signaling Pathways Requiring Vangl2 and Cyclic GMP

(2011) Roberta Buono et al.   STEM CELLS

Chemical treatment enhances skipping of a mutated exon in the dystrophin gene

(2011) Atsushi Nishida et al.   Nature Communications

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

(2010) Hong M. Moulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Abnormal distribution of inositol 1,4,5-trisphosphate receptors in human muscle can be related to altered calcium signals and gene expression in Duchenne dystrophy-derived cells

(2010) César Cárdenas et al.   FASEB JOURNAL

Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial

(2010) Janbernd Kirschner et al.   LANCET NEUROLOGY

Guanine Analogues Enhance Antisense Oligonucleotide-induced Exon Skipping in Dystrophin Gene In Vitro and In Vivo

(2010) Yihong Hu et al.   MOLECULAR THERAPY

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

(2010) Yoshitsugu Aoki et al.   MOLECULAR THERAPY

North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy

(2010) Elena Mazzone et al.   NEUROMUSCULAR DISORDERS

Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients

(2010) A.T.J.M. Helderman-van den Enden et al.   NEUROMUSCULAR DISORDERS

Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials

(2010) Virginia Arechavala-Gomeza et al.   NEUROMUSCULAR DISORDERS

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy

(2010) Beniamino Palmieri et al.   PEDIATRIC TRANSPLANTATION

Partial dysferlin reconstitution by adult murine mesoangioblasts is sufficient for full functional recovery in a murine model of dysferlinopathy

(2010) J Díaz-Manera et al.   Cell Death & Disease

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Comparison between gentamycin and exon skipping treatments to restore ryanodine receptor subtype 2 functions in mdx mouse duodenum myocytes

(2009) Fabrice Dabertrand et al.   EUROPEAN JOURNAL OF PHARMACOLOGY

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

(2009) Hans A. Heemskerk et al.   JOURNAL OF GENE MEDICINE

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

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

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy

(2009) Craig M. McDonald et al.   MUSCLE & NERVE

Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm

(2009) Philip Doran et al.   PROTEOMICS

Role of IGF-I in skeletal muscle mass maintenance

(2009) David R. Clemmons   TRENDS IN ENDOCRINOLOGY AND METABOLISM

Applications of metabolomics and proteomics to the mdx mouse model of Duchenne muscular dystrophy: lessons from downstream of the transcriptome

(2009) Julian L Griffin et al.   Genome Medicine

Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance

(2008) G. M. Buyse et al.   EUROPEAN HEART JOURNAL

A canine minidystrophin is functional and therapeutic in mdx mice

(2008) B Wang et al.   GENE THERAPY

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS