Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy

Halofuginone promotes satellite cell activation and survival in muscular dystrophies

(2016) Hila Barzilai-Tutsch et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Anti-fibrotic effect of pirfenidone in muscle derived-fibroblasts from Duchenne muscular dystrophy patients

(2016) Simona Zanotti et al.   LIFE SCIENCES

In vivoneutralization of IL-6 receptors ameliorates gastrointestinal dysfunction in dystrophin-deficientmdxmice

(2016) J. Manning et al.   NEUROGASTROENTEROLOGY AND MOTILITY

Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy

(2016) David G. Allen et al.   PHYSIOLOGICAL REVIEWS

The chaperone co-inducer BGP-15 alleviates ventilation-induced diaphragm dysfunction

(2016) Heba Salah et al.   Science Translational Medicine

Tadalafil Treatment Delays the Onset of Cardiomyopathy in Dystrophin‐Deficient Hearts

(2016) David W. Hammers et al.   Journal of the American Heart Association

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

The Pathogenesis and Therapy of Muscular Dystrophies

(2015) Simon Guiraud et al.   Annual Review of Genomics and Human Genetics

Altered ROS production, NF-κB activation and interleukin-6 gene expression induced by electrical stimulation in dystrophic mdx skeletal muscle cells

(2015) Carlos Henríquez-Olguín et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Pirfenidone for idiopathic pulmonary fibrosis: analysis of pooled data from three multinational phase 3 trials

(2015) Paul W. Noble et al.   EUROPEAN RESPIRATORY JOURNAL

Tamoxifen Inhibits TGF-β-Mediated Activation of Myofibroblasts by Blocking Non-Smad Signaling Through ERK1/2

(2015) Jon M. Carthy et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Excess TGF-β mediates muscle weakness associated with bone metastases in mice

(2015) David L Waning et al.   NATURE MEDICINE

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

(2015) Luca Bello et al.   NEUROLOGY

Dystrophin–glycoprotein complex regulates muscle nitric oxide production through mechanoregulation of AMPK signaling

(2015) Joanne F. Garbincius et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Inhibition of muscle fibrosis results in increases in both utrophin levels and the number of revertant myofibers in Duchenne muscular dystrophy

(2015) Oshrat Levi et al.   Oncotarget

Chronic Dosing with Membrane Sealant Poloxamer 188 NF Improves Respiratory Dysfunction in Dystrophic Mdx and Mdx/Utrophin-/- Mice

(2015) Bruce E. Markham et al.   PLoS One

Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy

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

Characterization of GLPG0492, a selective androgen receptor modulator, in a mouse model of hindlimb immobilization

(2014) Roland Blanqué et al.   BMC MUSCULOSKELETAL DISORDERS

Hyperactive Adverse Mechanical Stress Responses in Dystrophic Heart Are Coupled to Transient Receptor Potential Canonical 6 and Blocked by cGMP–Protein Kinase G Modulation

(2014) Kinya Seo et al.   CIRCULATION RESEARCH

Pharmacologic Management of Duchenne Muscular Dystrophy: Target Identification and Preclinical Trials

(2014) J. N. Kornegay et al.   ILAR JOURNAL

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

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

G.P.101

(2014) J. Milne et al.   NEUROMUSCULAR DISORDERS

Long-term administration of the TNF blocking drug Remicade (cV1q) to mdx mice reduces skeletal and cardiac muscle fibrosis, but negatively impacts cardiac function

(2014) N.V. Ermolova et al.   NEUROMUSCULAR DISORDERS

Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function

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

Prednisolone Attenuates Improvement of Cardiac and Skeletal Contractile Function and Histopathology by Lisinopril and Spironolactone in the mdx Mouse Model of Duchenne Muscular Dystrophy

(2014) Paul M. L. Janssen et al.   PLoS One

Diapocynin, a Dimer of the NADPH Oxidase Inhibitor Apocynin, Reduces ROS Production and Prevents Force Loss in Eccentrically Contracting Dystrophic Muscle

(2014) Hesham M. Ismail et al.   PLoS One

The small-molecule BGP-15 protects against heart failure and atrial fibrillation in mice

(2014) Geeta Sapra et al.   Nature Communications

NBD delivery improves the disease phenotype of the golden retriever model of Duchenne muscular dystrophy

(2014) Joe N Kornegay et al.   Skeletal Muscle

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

Pharmacological prospects in the treatment of Duchenne muscular dystrophy

(2013) Urs T. Ruegg   CURRENT OPINION IN NEUROLOGY

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

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

Suramin attenuates dystrophin-deficient cardiomyopathy in themdxmouse model of duchenne muscular dystrophy

(2013) Drielen De Oliveira Moreira et al.   MUSCLE & NERVE

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

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

Imatinib attenuates severe mouse dystrophy and inhibits proliferation and fibrosis-marker expression in muscle mesenchymal progenitors

(2013) Takahito Ito et al.   NEUROMUSCULAR DISORDERS

GLPG0492, a novel selective androgen receptor modulator, improves muscle performance in the exercised-mdx mouse model of muscular dystrophy

(2013) Anna Cozzoli et al.   PHARMACOLOGICAL RESEARCH

Emerging drugs for Duchenne muscular dystrophy

(2012) Vinod Malik et al.   EXPERT OPINION ON EMERGING DRUGS

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

Contribution of oxidative stress to pathology in diaphragm and limb muscles with Duchenne muscular dystrophy

(2012) Jong-Hee Kim et al.   JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY

Early corticosteroid treatment in 4 duchenne muscular dystrophy patients: 14-year follow-up

(2012) Luciano Merlini et al.   MUSCLE & NERVE

Suramin affects metalloproteinase-9 activity and increases beta-dystroglycan levels in the diaphragm of the dystrophin-deficientmdxMOUSE

(2012) Ana Paula Tiemi Taniguti et al.   MUSCLE & NERVE

Hsp72 preserves muscle function and slows progression of severe muscular dystrophy

(2012) Stefan M. Gehrig et al.   NATURE

Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial

(2012) D. M. Escolar et al.   NEUROLOGY

Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy

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

Recombinant MG53 Protein Modulates Therapeutic Cell Membrane Repair in Treatment of Muscular Dystrophy

(2012) N. Weisleder et al.   Science Translational Medicine

Long-Term Blocking of Calcium Channels in mdx Mice Results in Differential Effects on Heart and Skeletal Muscle

(2011) Louise H. Jørgensen et al.   AMERICAN JOURNAL OF PATHOLOGY

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

Dysferlin, Annexin A1, and Mitsugumin 53 Are Upregulated in Muscular Dystrophy and Localize to Longitudinal Tubules of the T-System With Stretch

(2011) Leigh B. Waddell et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Melatonin improves muscle function of the dystrophic mdx5Cv mouse, a model for Duchenne muscular dystrophy

(2011) Youssef Hibaoui et al.   JOURNAL OF PINEAL RESEARCH

Improvement of cardiac contractile function by peptide-based inhibition of NF-κB in the utrophin/dystrophin-deficient murine model of muscular dystrophy

(2011) Dawn A Delfín et al.   Journal of Translational Medicine

Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy

(2011) Angela Zimmerman et al.   MUSCLE & NERVE

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

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

Systemic delivery of NEMO binding domain/IKKγ inhibitory peptide to young mdx mice improves dystrophic skeletal muscle histopathology

(2011) Daniel P. Reay et al.   NEUROBIOLOGY OF DISEASE

N-Acetylcysteine treatment of dystrophic mdx mice results in protein thiol modifications and inhibition of exercise induced myofibre necrosis

(2011) Jessica R. Terrill et al.   NEUROMUSCULAR DISORDERS

Chronic Losartan Administration Reduces Mortality and Preserves Cardiac but Not Skeletal Muscle Function in Dystrophic Mice

(2011) Lawrence T. Bish et al.   PLoS One

Effects of a Novel Selective Androgen Receptor Modulator on Dexamethasone-Induced and Hypogonadism-Induced Muscle Atrophy

(2010) Amanda Jones et al.   ENDOCRINOLOGY

Halofuginone inhibits Smad3 phosphorylation via the PI3K/Akt and MAPK/ERK pathways in muscle cells: Effect on myotube fusion

(2010) Suzy Roffe et al.   EXPERIMENTAL CELL RESEARCH

Regulation and dysregulation of fibrosis in skeletal muscle

(2010) Antonio L. Serrano et al.   EXPERIMENTAL CELL RESEARCH

Overexpression of SERCA1a in the mdx Diaphragm Reduces Susceptibility to Contraction-Induced Damage

(2010) Kevin J. Morine et al.   HUMAN GENE THERAPY

Administration of a soluble activin type IIB receptor promotes skeletal muscle growth independent of fiber type

(2010) Samuel M. Cadena et al.   JOURNAL OF APPLIED PHYSIOLOGY

Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management

(2010) Richard T. Moxley et al.   JOURNAL OF CHILD NEUROLOGY

Golgi and sarcolemmal neuronal NOS differentially regulate contraction-induced fatigue and vasoconstriction in exercising mouse skeletal muscle

(2010) Justin M. Percival et al.   JOURNAL OF CLINICAL INVESTIGATION

Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs

(2010) DeWayne Townsend et al.   JOURNAL OF CLINICAL INVESTIGATION

Melatonin treatment normalizes plasma pro-inflammatory cytokines and nitrosative/oxidative stress in patients suffering from Duchenne muscular dystrophy

(2010) Mariam Chahbouni et al.   JOURNAL OF PINEAL RESEARCH

Prevention of muscle fibrosis and myonecrosis inmdxmice by suramin, a TGF-β1 blocker

(2010) Ana Paula Tiemi Taniguti et al.   MUSCLE & NERVE

Tamoxifen is associated with lower mortality of encapsulating peritoneal sclerosis: results of the Dutch Multicentre EPS Study

(2010) M. R. Korte et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Debio-025 is more effective than prednisone in reducing muscular pathology in mdx mice

(2010) Erin R. Wissing et al.   NEUROMUSCULAR DISORDERS

Skeletal Muscle NADPH Oxidase Is Increased and Triggers Stretch-Induced Damage in the mdx Mouse

(2010) Nicholas P. Whitehead et al.   PLoS One

Leaky RyR2 trigger ventricular arrhythmias in Duchenne muscular dystrophy

(2010) J. Fauconnier et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sildenafil reverses cardiac dysfunction in the mdx mouse model of Duchenne muscular dystrophy

(2010) C. M. Adamo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Emerging strategies for cell and gene therapy of the muscular dystrophies

(2009) Lindsey A. Muir et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Automated drug screening with contractile muscle tissue engineered from dystrophic myoblasts

(2009) Herman Vandenburgh et al.   FASEB JOURNAL

Imatinib attenuates skeletal muscle dystrophy in mdx mice

(2009) Ping Huang et al.   FASEB JOURNAL

The antifibrotic drug halofuginone inhibits proliferation and collagen production by human leiomyoma and myometrial smooth muscle cells

(2009) Meagan M. Grudzien et al.   FERTILITY AND STERILITY

Multiple pathological events in exercised dystrophic mdx mice are targeted by pentoxifylline: outcome of a large array of in vivo and ex vivo tests

(2009) Rosa Burdi et al.   JOURNAL OF APPLIED PHYSIOLOGY

Imatinib mesylate ameliorates the dystrophic phenotype in exercised mdx mice

(2009) João Carlos da Silva Bizario et al.   JOURNAL OF NEUROIMMUNOLOGY

Hypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic muscle

(2009) Andrew M Bellinger et al.   NATURE MEDICINE

l-Arginine Decreases Inflammation and Modulates the Nuclear Factor-κB/Matrix Metalloproteinase Cascade in Mdx Muscle Fibers

(2008) Karim Hnia et al.   AMERICAN JOURNAL OF PATHOLOGY

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

(2008) Kathryn R. Wagner et al.   ANNALS OF NEUROLOGY

Regulation of muscle mass by growth hormone and IGF-I

(2008) C P Velloso   BRITISH JOURNAL OF PHARMACOLOGY

Investigation of Debio 025, a cyclophilin inhibitor, in the dystrophic mdx mouse, a model for Duchenne muscular dystrophy

(2008) J Reutenauer et al.   BRITISH JOURNAL OF PHARMACOLOGY

Imatinib mesylate attenuates fibrosis in coxsackievirus b3-induced chronic myocarditis

(2008) Carola Leipner et al.   CARDIOVASCULAR RESEARCH

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

Fibrinogen drives dystrophic muscle fibrosis via a TGF /alternative macrophage activation pathway

(2008) B. Vidal et al.   GENES & DEVELOPMENT

N-Acetylcysteine ameliorates skeletal muscle pathophysiology inmdxmice

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

Sarcolemma-localized nNOS is required to maintain activity after mild exercise

(2008) Yvonne M. Kobayashi et al.   NATURE

Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy

(2008) Douglas P Millay et al.   NATURE MEDICINE

Sildenafil and cardiomyocyte-specific cGMP signaling prevent cardiomyopathic changes associated with dystrophin deficiency

(2008) M. Khairallah et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Connective tissue growth factor is overexpressed in muscles of human muscular dystrophy

(2007) Guilian Sun et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES