Clinical utility of serum biomarkers in Duchenne muscular dystrophy

Comparative mass spectrometric and immunoassay-based proteome analysis in serum of Duchenne muscular dystrophy patients

(2016) Stijn Oonk et al.   Proteomics Clinical Applications

Proteomic methods for biomarker discovery and validation. Are we there yet?

(2015) Yetrib Hathout   Expert Review of Proteomics

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

(2015) Jérémy Rouillon et al.   HUMAN MOLECULAR GENETICS

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus

(2015) Yongping Yue et al.   HUMAN MOLECULAR GENETICS

Comparative Label-Free Mass Spectrometric Analysis of Mildly versus Severely Affected mdx Mouse Skeletal Muscles Identifies Annexin, Lamin, and Vimentin as Universal Dystrophic Markers

(2015) Ashling Holland et al.   MOLECULES

Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study

(2015) Luca Bello et al.   NEUROLOGY

Response to: Evaluation of the serum matrix metalloproteinase-9 as a biomarker for monitoring disease progression in Duchenne muscular dystrophy

(2015) Pietro Spitali et al.   NEUROMUSCULAR DISORDERS

204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24–26 January 2014, Naarden, The Netherlands

(2015) Alessandra Ferlini et al.   NEUROMUSCULAR DISORDERS

Evaluation of the serum matrix metalloproteinase-9 as a biomarker for monitoring disease progression in Duchenne muscular dystrophy

(2015) Aleksandar Zocevic 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

Glucocorticoids enhance muscle endurance and ameliorate Duchenne muscular dystrophy through a defined metabolic program

(2015) Alexander Morrison-Nozik et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

(2015) Yetrib Hathout et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics

(2015) Anna M. L. Coenen-Stass et al.   Scientific Reports

TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy

(2015) Alyson A. Fiorillo et al.   Cell Reports

Gene therapy for Duchenne muscular dystrophy

(2015) Julian Ramos et al.   Expert Opinion on Orphan Drugs

Elusive sources of variability of dystrophin rescue by exon skipping

(2015) Maria Candida Vila et al.   Skeletal Muscle

Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients

(2014) Laurence Jeanson-Leh et al.   AMERICAN JOURNAL OF PATHOLOGY

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

EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal

(2014) Asher Mullard   NATURE BIOTECHNOLOGY

Longitudinal measurements of MRI-T2 in boys with Duchenne muscular dystrophy: Effects of age and disease progression

(2014) R.J. Willcocks et al.   NEUROMUSCULAR DISORDERS

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

(2014) Jeremy Rouillon et al.   NEUROMUSCULAR DISORDERS

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

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

Beyond antibodies: New affinity reagents to unlock the proteome

(2014) Bridget Lollo et al.   PROTEOMICS

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

(2014) Qi-long Lu et al.   Molecular Therapy-Nucleic Acids

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

Identification of Disease Specific Pathways Usingin VivoSILAC Proteomics in Dystrophin DeficientmdxMouse

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

Magnetic resonance imaging in duchenne muscular dystrophy: Longitudinal assessment of natural history over 18 months

(2013) Kieren G. Hollingsworth et al.   MUSCLE & NERVE

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

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

THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

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

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

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

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy

(2013) Irina T. Zaharieva 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

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

Muscle histology vs MRI in Duchenne muscular dystrophy

(2011) M. Kinali et al.   NEUROLOGY

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

(2011) D. M. Escolar et al.   NEUROLOGY

Serum matrix metalloproteinase-9 (MMP-9) as a biomarker for monitoring disease progression in Duchenne muscular dystrophy (DMD)

(2011) V.D. Nadarajah et al.   NEUROMUSCULAR DISORDERS

Advances in human proteomics at high scale with the SOMAscan proteomics platform

(2011) Larry Gold et al.   New Biotechnology

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

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

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Reliability of the North Star Ambulatory Assessment in a multicentric setting

(2009) E.S. Mazzone et al.   NEUROMUSCULAR DISORDERS