Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy

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

(2012) Luciano Merlini et al.   MUSCLE & NERVE

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

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

Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy

(2012) L. Bello et al.   NEUROLOGY

Pharmaceutical therapies to recode nonsense mutations in inherited diseases

(2012) Hui-Ling Rose Lee et al.   PHARMACOLOGY & THERAPEUTICS

Glucocorticoid Analogues: Potential Therapeutic Alternatives for Treating Inflammatory Muscle Diseases

(2012) Erica K.M. Reeves et al.   Endocrine Metabolic & Immune Disorders-Drug Targets

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

(2011) K. Anthony et al.   BRAIN

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

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

Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

(2011) Yadong Zhang et al.   HUMAN GENE THERAPY

A randomized controlled clinical trial of growth hormone in amyotrophic lateral sclerosis: clinical, neuroimaging, and hormonal results

(2011) Francesco Saccà et al.   JOURNAL OF NEUROLOGY

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

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

(2011) Sebahattin Cirak et al.   MOLECULAR THERAPY

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

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

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Open-Label Trial of Recombinant Human Insulin-like Growth Factor 1/Recombinant Human Insulin-like Growth Factor Binding Protein 3 in Myotonic Dystrophy Type 1

(2010) Chad R. Heatwole et al.   ARCHIVES OF NEUROLOGY

Classic glucocorticoids versus non-steroidal glucocorticoid receptor modulators: Survival of the fittest regulator of the immune system?

(2010) Karolien De Bosscher et al.   BRAIN BEHAVIOR AND IMMUNITY

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

Effects of Insulin-Like Growth Factor (IGF)-I/IGF-Binding Protein-3 Treatment on Glucose Metabolism and Fat Distribution in Human Immunodeficiency Virus-Infected Patients with Abdominal Obesity and Insulin Resistance

(2010) Madhu N. Rao et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial

(2010) Janbernd Kirschner et al.   LANCET NEUROLOGY

Effect of creatine monohydrate in improving cellular energetics and muscle strength in ambulatory Duchenne muscular dystrophy patients: a randomized, placebo-controlled 31P MRS study

(2010) Bidisha Banerjee et al.   MAGNETIC RESONANCE IMAGING

Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

(2010) Joe N Kornegay et al.   MOLECULAR THERAPY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Inhibiting Myostatin with Follistatin Improves the Success of Myoblast Transplantation in Dystrophic Mice

(2009) Basma F. Benabdallah et al.   CELL TRANSPLANTATION

Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

(2009) Igor Nudelman et al.   JOURNAL OF MEDICINAL CHEMISTRY

Delayed Diagnosis in Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

(2009) Emma Ciafaloni et al.   JOURNAL OF PEDIATRICS

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Lack of Functional Benefit with Glutamine versus Placebo in Duchenne Muscular Dystrophy: A Randomized Crossover Trial

(2009) Elise Mok et al.   PLoS One

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

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

Differential Antagonism of Activin, Myostatin and Growth and Differentiation Factor 11 by Wild-Type and Mutant Follistatin

(2008) Alan L. Schneyer et al.   ENDOCRINOLOGY

RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology

(2008) Mohammad M. Ghahramani Seno et al.   HUMAN MOLECULAR GENETICS

Subcutaneous IGF-1 is not beneficial in 2-year ALS trial

(2008) E. J. Sorenson et al.   NEUROLOGY

Deflazacort Use in Duchenne Muscular Dystrophy: An 8-Year Follow-Up

(2008) Sylvie Houde et al.   PEDIATRIC NEUROLOGY

Muscle-specific overexpression of IGF-I improves E-C coupling in skeletal muscle fibers from dystrophic mdx mice

(2007) Jonathan D. Schertzer et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice

(2007) Masashi Nakatani et al.   FASEB JOURNAL