Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies

Affinity proteomics within rare diseases: a BIO-NMD study for blood biomarkers of muscular dystrophies

(2014) B. Ayoglu et al.   EMBO Molecular Medicine

Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients

(2014) Yetrib Hathout et al.   HUMAN MOLECULAR GENETICS

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

Proteomics profiling of urine reveals specific titin fragments as biomarkers of Duchenne muscular dystrophy

(2014) Jeremy Rouillon et al.   NEUROMUSCULAR DISORDERS

Dysfunctional Muscle and Liver Glycogen Metabolism in mdx Dystrophic Mice

(2014) David I. Stapleton et al.   PLoS One

Dystrophin deficiency leads to disturbance of LAMP1-vesicle-associated protein secretion

(2013) Stephanie Duguez et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Newborn bloodspot screening for Duchenne Muscular Dystrophy: 21 years experience in Wales (UK)

(2013) Stuart J Moat et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Prolonged Gene Expression in Muscle Is Achieved Without Active Immune Tolerance Using MicrorRNA 142.3p-Regulated rAAV Gene Transfer

(2013) Florence Boisgerault et al.   HUMAN GENE THERAPY

Comparison of dixon and T1-weighted MR methods to assess the degree of fat infiltration in duchenne muscular dystrophy patients

(2013) Beatrijs H. Wokke et al.   JOURNAL OF MAGNETIC RESONANCE IMAGING

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   MUSCLE & NERVE

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

Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover

(2013) Thomas C. Roberts et al.   NUCLEIC ACIDS RESEARCH

Distinctive Serum miRNA Profile in Mouse Models of Striated Muscular Pathologies

(2013) Nicolas Vignier et al.   PLoS One

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

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

Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C

(2012) Serge Herson et al.   BRAIN

Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies

(2012) Joe N. Kornegay et al.   MAMMALIAN GENOME

Enhancing Muscle Membrane Repair by Gene Delivery of MG53 Ameliorates Muscular Dystrophy and Heart Failure in δ-Sarcoglycan-deficient Hamsters

(2012) Bo He et al.   MOLECULAR THERAPY

Innovative methods to assess upper limb strength and function in non-ambulant Duchenne patients

(2012) L. Servais et al.   NEUROMUSCULAR DISORDERS

Exploration of Lipid Metabolism in Relation with Plasma Membrane Properties of Duchenne Muscular Dystrophy Cells: Influence of L-Carnitine

(2012) Françoise Le Borgne et al.   PLoS One

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

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

miRNAs as serum biomarkers for Duchenne muscular dystrophy

(2011) Davide Cacchiarelli et al.   EMBO Molecular Medicine

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

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

(2011) HaiFang Yin et al.   MOLECULAR THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Endpoint measures in the mdx mouse relevant for muscular dystrophy pre-clinical studies

(2011) Yvonne M. Kobayashi et al.   NEUROMUSCULAR DISORDERS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

(2010) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

(2010) William Lostal et al.   HUMAN MOLECULAR GENETICS

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers†

(2009) Natee Jearawiriyapaisarn et al.   CARDIOVASCULAR RESEARCH

A simple protocol for assessing inter-trial and inter-examiner reliability for two noninvasive measures of limb muscle strength

(2009) C. George Carlson et al.   JOURNAL OF NEUROSCIENCE METHODS

Measurement of the Functional Status of Patients with Different Types of Muscular Dystrophy

(2009) Yi-Jing Lue et al.   KAOHSIUNG JOURNAL OF MEDICAL SCIENCES

A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells

(2009) Richard H Smith et al.   MOLECULAR THERAPY

Use of evans blue dye to compare limb muscles in exercised young and oldmdxmice

(2009) Christine I. Wooddell et al.   MUSCLE & NERVE

Cardiac ankyrin repeat protein is a marker of skeletal muscle pathological remodelling

(2008) Lydie Laure et al.   FEBS Journal

MG53 nucleates assembly of cell membrane repair machinery

(2008) Chuanxi Cai et al.   NATURE CELL BIOLOGY

Myomesin 3, a Novel Structural Component of the M-band in Striated Muscle

(2007) Roman Schoenauer et al.   JOURNAL OF MOLECULAR BIOLOGY