Emerging drugs for Duchenne muscular dystrophy

Evidence-based path to newborn screening for duchenne muscular dystrophy

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

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

Effects of Naproxcinod on Blood Pressure in Patients With Osteoarthritis

(2011) William B. White et al.   AMERICAN JOURNAL OF CARDIOLOGY

Melatonin treatment counteracts the hyperoxidative status in erythrocytes of patients suffering from Duchenne muscular dystrophy

(2011) Mariam Chahbouni et al.   CLINICAL BIOCHEMISTRY

Pirfenidone

(2011) Natalie J. Carter   DRUGS

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

(2011) M. Wilschanski et al.   EUROPEAN RESPIRATORY JOURNAL

Factors Associated With Treatment Response to Etanercept in Juvenile Idiopathic Arthritis

(2011) Marieke H. Otten et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

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

Blood Pressure Effects of Naproxcinod in Hypertensive Patients

(2011) Raymond Townsend et al.   Journal of Clinical Hypertension

Exacerbation of pathology by oxidative stress in respiratory and locomotor muscles with Duchenne muscular dystrophy

(2011) John M. Lawler   JOURNAL OF PHYSIOLOGY-LONDON

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

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

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(2011) Jennifer Peterson et al.   MOLECULAR MEDICINE

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

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

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

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

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

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

Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse

(2011) Jonathon M. Tinsley et al.   PLoS One

Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening

(2011) Catherine Moorwood et al.   PLoS One

Efficacy, safety, and effects on blood pressure of naproxcinod 750 mg twice daily compared with placebo and naproxen 500 mg twice daily in patients with osteoarthritis of the hip: A randomized, double-blind, parallel-group, multicenter study

(2010) Christoph Baerwald et al.   ARTHRITIS AND RHEUMATISM

MicroRNAs Involved in Molecular Circuitries Relevant for the Duchenne Muscular Dystrophy Pathogenesis Are Controlled by the Dystrophin/nNOS Pathway

(2010) Davide Cacchiarelli et al.   Cell Metabolism

Clinical Uses of Melatonin: Evaluation of Human Trials

(2010) E.J. Sanchez-Barcelo et al.   CURRENT MEDICINAL CHEMISTRY

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

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

Regulation of Muscle Mass by Follistatin and Activins

(2010) Se-Jin Lee et al.   MOLECULAR ENDOCRINOLOGY

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

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

Blockade of TNF in vivo using cV1q antibody reduces contractile dysfunction of skeletal muscle in response to eccentric exercise in dystrophic mdx and normal mice

(2010) A.T. Piers et al.   NEUROMUSCULAR DISORDERS

Stem cells to treat muscular dystrophies – Where are we?

(2010) Jinhong Meng et al.   NEUROMUSCULAR DISORDERS

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

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

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

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

(2010) A. R. Amenta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficacy and Safety of Naproxcinod in Patients with Osteoarthritis of the Knee: A 53-Week Prospective Randomized Multicenter Study

(2010) Thomas J. Schnitzer et al.   SEMINARS IN ARTHRITIS AND RHEUMATISM

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Pharmaceuticals Targeting Nonsense Mutations in Genetic Diseases

(2009) Steven M. Rowe et al.   BIODRUGS

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

Pain and activity limitations in children with Duchenne or Becker muscular dystrophy

(2009) Kathy Zebracki et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Imatinib attenuates skeletal muscle dystrophy in mdx mice

(2009) Ping Huang et al.   FASEB JOURNAL

Imatinib mesylate ameliorates the dystrophic phenotype in exercised mdx mice

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

Endomysial Fibrosis in Duchenne Muscular Dystrophy: A Marker of Poor Outcome Associated With Macrophage Alternative Activation

(2009) Isabelle Desguerre et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

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

Development of a Nitric Oxide-Releasing Analogue of the Muscle Relaxant Guaifenesin for Skeletal Muscle Satellite Cell Myogenesis

(2009) Guqi Wang et al.   MOLECULAR PHARMACEUTICS

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

(2009) Louise R. Rodino-Klapac et al.   MUSCLE & NERVE

A nitric oxide/cysteine interaction mediates the activation of soluble guanylate cyclase

(2009) N. B. Fernhoff et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Functional resolution of fibrosis in mdx mouse dystrophic heart and skeletal muscle by halofuginone

(2008) Kyla D. Huebner et al.   AMERICAN JOURNAL OF PHYSIOLOGY-HEART AND CIRCULATORY PHYSIOLOGY

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

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

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

Subcutaneous injection, from birth, of epigallocatechin-3-gallate, a component of green tea, limits the onset of muscular dystrophy in mdx mice: a quantitative histological, immunohistochemical and electrophysiological study

(2008) Yoshiko Nakae et al.   HISTOCHEMISTRY AND CELL BIOLOGY

Skeletal Muscle Diseases, Inflammation, and NF-κB Signaling: Insights and Opportunities for Therapeutic Intervention

(2008) Jennifer M. Peterson et al.   INTERNATIONAL REVIEWS OF IMMUNOLOGY

Sustained Soluble Guanylate Cyclase Stimulation Offsets Nitric-Oxide Synthase Inhibition to Restore Acute Cardiac Modulation by Sildenafil

(2008) T. Nagayama et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

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

Prevention of muscle fibrosis and improvement in muscle performance in the mdx mouse by halofuginone

(2008) Tidhar Turgeman et al.   NEUROMUSCULAR DISORDERS

Etanercept Treatment for Children and Adolescents with Plaque Psoriasis

(2008) Amy S. Paller et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

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

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