Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy

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

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

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

Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy

(2018) Mark A. Aminzadeh et al.   Stem Cell Reports

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

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

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

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

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

Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy

(2016) Robert C. Griggs et al.   NEUROLOGY

Modifier genes and their effect on Duchenne muscular dystrophy

(2015) Andy H. Vo et al.   CURRENT OPINION IN NEUROLOGY

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

Border between natural product and drug: Comparison of the related benzoquinones idebenone and coenzyme Q10

(2015) Nuri Gueven et al.   Redox Biology

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

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

Newborn bloodspot screening for Duchenne Muscular Dystrophy: 21 years experience in Wales (UK)

(2013) Stuart J Moat et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Glucocorticoid Treatment for the Prevention of Scoliosis in Children with Duchenne Muscular Dystrophy: Long-Term Follow-up

(2013) David E Lebel et al.   JOURNAL OF BONE AND JOINT SURGERY-AMERICAN VOLUME

Corticosteroids in Duchenne muscular dystrophy: Major variations in practice

(2013) Robert C. Griggs et al.   MUSCLE & NERVE

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe

(2013) Erik K. Henricson et al.   MUSCLE & NERVE

Evidence-based path to newborn screening for duchenne muscular dystrophy

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

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

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

Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

(2008) Paul T. Martin et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY