Therapeutic developments for Duchenne muscular dystrophy

Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial

(2019) Michael Taylor et al.   NEUROLOGY

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy

(2019) Panteleimon D. Mavroudis et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients

(2018) Christian Leborgne et al.   CELLULAR IMMUNOLOGY

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

(2018) Craig M McDonald et al.   LANCET

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

(2018) Silvana M.G. Jirka et al.   MOLECULAR THERAPY

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Deflazacort vs prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post hoc analysis from the ACT DMD trial

(2018) Perry B Shieh et al.   MUSCLE & NERVE

Why dystrophin quantification is key in the eteplirsen saga

(2018) Annemieke Aartsma-Rus et al.   Nature Reviews Neurology

Eteplirsen treatment for Duchenne muscular dystrophy

(2018) Jay S. Charleston et al.   NEUROLOGY

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

(2018) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017

(2018) Volker Straub et al.   NEUROMUSCULAR DISORDERS

Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

(2018) Hirofumi Komaki et al.   Science Translational Medicine

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes

(2018) Eric P. Hoffman et al.   STEROIDS

Therapeutic Mechanisms of Glucocorticoids

(2018) Jolien Vandewalle et al.   TRENDS IN ENDOCRINOLOGY AND METABOLISM

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

(2018) Craig M. McDonald et al.   Annals of Clinical and Translational Neurology

CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo

(2018) Benjamin L. Duchêne et al.   MOLECULAR THERAPY

DMD CLINICAL THERAPIES I

(2018) K. Wagner et al.   NEUROMUSCULAR DISORDERS

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

(2018) Laurie S. Conklin et al.   PHARMACOLOGICAL RESEARCH

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

(2018) Michael J. Gait et al.   Nucleic Acid Therapeutics

A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe

(2018) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

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

Reduced serum myostatin concentrations associated with genetic muscle disease progression

(2017) Peter M. Burch et al.   JOURNAL OF NEUROLOGY

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

(2017) Craig M McDonald et al.   LANCET

Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5 ch and AAV1

(2017) Anna Majowicz et al.   MOLECULAR THERAPY

Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides

(2017) Naoki Iwamoto et al.   NATURE BIOTECHNOLOGY

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

(2017) Ronald G. Victor et al.   NEUROLOGY

Stunning pharmacological properties of DS-5141b, an antisense oligonucleotide consisting of 2'-O,4'-C-ethylene-bridged nucleic acids and 2'-O-methyl RNA, on dystrophin mRNA exon skipping

(2017) K. Takaishi et al.   NEUROMUSCULAR DISORDERS

A Phase I, single- and repeated-dose study of TAS-205, a novel inhibitor of hematopoietic prostaglandin D synthase, in patients with Duchenne muscular dystrophy

(2017) H. Komaki et al.   NEUROMUSCULAR DISORDERS

Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy

(2017) Thomas Meier et al.   NEUROMUSCULAR DISORDERS

Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: results of a two-year open-label extension trial

(2017) Subha V. Raman et al.   Orphanet Journal of Rare Diseases

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

(2017) Yusuke Echigoya et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

(2017) Virginie Mariot et al.   Nature Communications

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

(2017) Ngoc Lu-Nguyen et al.   Molecular Therapy-Nucleic Acids

A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy and its humanized equivalent, domagrozumab (PF-06252616), increases muscle volume in cynomolgus monkeys

(2017) Michael St. Andre et al.   Skeletal Muscle

The golden retriever model of Duchenne muscular dystrophy

(2017) Joe N. Kornegay   Skeletal Muscle

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

(2016) Courtney S. Young et al.   Cell Stem Cell

Intra‐arterial transplantation of HLA‐matched donor mesoangioblasts in Duchenne muscular dystrophy

(2016) Giulio Cossu et al.   EMBO Molecular Medicine

The importance of genetic diagnosis for Duchenne muscular dystrophy

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

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

(2016) Volker Straub et al.   LANCET NEUROLOGY

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial

(2016) Craig Campbell et al.   MUSCLE & NERVE

Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy

(2016) Craig M. McDonald et al.   NEUROMUSCULAR DISORDERS

Histological effects of givinostat in boys with Duchenne muscular dystrophy

(2016) Paolo Bettica et al.   NEUROMUSCULAR DISORDERS

Exon Snipping in Duchenne Muscular Dystrophy

(2016) Dwi U. Kemaladewi et al.   TRENDS IN MOLECULAR MEDICINE

Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contractures

(2016) Joe N. Kornegay et al.   Skeletal Muscle

Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study

(2016) Patricia Hafner et al.   PLoS One

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

(2016) Valeria Ricotti et al.   PLoS One

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells

(2016) Ignazio Maggio et al.   Scientific Reports

A Review of Nebivolol Pharmacology and Clinical Evidence

(2015) Justin Fongemie et al.   DRUGS

Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy

(2015) G. Cossu et al.   EMBO Molecular Medicine

Cell therapy in muscular dystrophies: many promises in mice and dogs, few facts in patients

(2015) Daniel Skuk et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

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

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

Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

(2015) Gunnar M Buyse et al.   LANCET

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

(2015) Jerry R Mendell et al.   MOLECULAR THERAPY

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

Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy

(2015) Tim Cheetham et al.   NEUROPEDIATRICS

A prudent path forward for genomic engineering and germline gene modification

(2015) D. Baltimore et al.   SCIENCE

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

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

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

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

Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy

(2014) Doris G. Leung et al.   ANNALS OF NEUROLOGY

Effect of sildenafil on skeletal and cardiac muscle in Becker muscular dystrophy

(2014) Nanna Witting et al.   ANNALS OF NEUROLOGY

Myostatin and the skeletal muscle atrophy and hypertrophy signaling pathways

(2014) J. Rodriguez et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Therapeutic suppression of premature termination codons: Mechanisms and clinical considerations (Review)

(2014) JOHN KARIJOLICH et al.   INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE

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

PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy

(2014) M. D. Nelson et al.   NEUROLOGY

Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function

(2014) Rebecca L. Terry et al.   PLoS One

Adeno-associated viral vectors do not efficiently target muscle satellite cells

(2014) Andrea LH Arnett et al.   Molecular Therapy-Methods & Clinical Development

The Anticancer Drug Tamoxifen Counteracts the Pathology in a Mouse Model of Duchenne Muscular Dystrophy

(2013) Olivier M. Dorchies et al.   AMERICAN JOURNAL OF PATHOLOGY

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(2013) Silvia Consalvi et al.   MOLECULAR MEDICINE

Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates

(2013) Louis G. Chicoine et al.   MOLECULAR THERAPY

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

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

Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice

(2013) Silvana M.G. Jirka et al.   Nucleic Acid Therapeutics

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency

(2013) HaiFang Yin et al.   Molecular Therapy-Nucleic Acids

Vascular-targeted therapies for Duchenne muscular dystrophy

(2013) James P Ennen et al.   Skeletal Muscle

Quantitative evaluation of the beneficial effects in the mdx mouse of epigallocatechin gallate, an antioxidant polyphenol from green tea

(2012) Yoshiko Nakae et al.   HISTOCHEMISTRY AND CELL BIOLOGY

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

(2012) Adeline Vulin et al.   MOLECULAR THERAPY

The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy

(2012) A. M. Sbrocchi et al.   OSTEOPOROSIS INTERNATIONAL

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

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

The Paradox of Muscle Hypertrophy in Muscular Dystrophy

(2012) Joe N. Kornegay et al.   Physical Medicine and Rehabilitation Clinics of North America

Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy

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

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

Targeting the Activin Type IIB Receptor to Improve Muscle Mass and Function in the mdx Mouse Model of Duchenne Muscular Dystrophy

(2011) Emidio E. Pistilli et al.   AMERICAN JOURNAL OF PATHOLOGY

Follistatin Improves Skeletal Muscle Healing after Injury and Disease through an Interaction with Muscle Regeneration, Angiogenesis, and Fibrosis

(2011) Jinhong Zhu et al.   AMERICAN JOURNAL OF PATHOLOGY

Membrane Sealant Poloxamer P188 Protects Against Isoproterenol Induced Cardiomyopathy in Dystrophin Deficient Mice

(2011) Christopher F Spurney et al.   BMC Cardiovascular Disorders

Long-Term Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Golden Retriever Muscular Dystrophy

(2011) Lawrence T. Bish et al.   HUMAN GENE THERAPY

CINRG pilot trial of coenzyme Q10 in steroid-treated duchenne muscular dystrophy

(2011) Christopher F. Spurney et al.   MUSCLE & NERVE

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Antibody-Directed Myostatin Inhibition Improves Diaphragm Pathology in Young but not Adult Dystrophic mdx Mice

(2010) Kate T. Murphy et al.   AMERICAN JOURNAL OF PATHOLOGY

Carvedilol can Prevent Cardiac Events in Duchenne Muscular Dystrophy

(2010) Tsuyoshi Matsumura et al.   INTERNAL MEDICINE

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

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

(2009) Natee Jearawiriyapaisarn et al.   CARDIOVASCULAR RESEARCH

Flavocoxid counteracts muscle necrosis and improves functional properties in mdx mice: A comparison study with methylprednisolone

(2009) Sonia Messina et al.   EXPERIMENTAL NEUROLOGY

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates

(2009) J. Kota et al.   Science Translational 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