Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy

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

A new era in the management of Duchenne muscular dystrophy

(2018) Rudolf Korinthenberg   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy – a literature review

(2017) Ingrid E. C. Verhaart et al.   Orphanet Journal of Rare Diseases

The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

(2017) S. Ryder et al.   Orphanet Journal of Rare Diseases

Global Consensus Recommendations on Prevention and Management of Nutritional Rickets

(2016) Craig F. Munns et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Histological effects of givinostat in boys with Duchenne muscular dystrophy

(2016) Paolo Bettica et al.   NEUROMUSCULAR DISORDERS

Twenty-Four Hour Noninvasive Ventilation in Duchenne Muscular Dystrophy: A Safe Alternative to Tracheostomy

(2016) Douglas A McKim et al.   Canadian Respiratory Journal

Clinical phenotypes as predictors of the outcome of skipping aroundDMDexon 45

(2015) Andrew R. Findlay et al.   ANNALS OF NEUROLOGY

The Pathogenesis and Therapy of Muscular Dystrophies

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

Contemporary Cardiac Issues in Duchenne Muscular Dystrophy: Table.

(2015) Elizabeth M. McNally et al.   CIRCULATION

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

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

Cognitive and Neurobehavioral Profile in Boys With Duchenne Muscular Dystrophy

(2015) Rudaina Banihani et al.   JOURNAL OF CHILD NEUROLOGY

Duchenne Muscular Dystrophy: From Diagnosis to Therapy

(2015) Maria Falzarano et al.   MOLECULES

Resistance strength training exercise in children with spinal muscular atrophy

(2015) Aga Lewelt et al.   MUSCLE & NERVE

Different types of upper extremity exercise training in Duchenne muscular dystrophy: Effects on functional performance, strength, endurance, and ambulation

(2015) Ipek Alemdaroğlu et al.   MUSCLE & NERVE

Spinal Muscular Atrophy

(2015) Stephen J. Kolb et al.   NEUROLOGIC CLINICS

Quality improvement in neurology: Table

(2015) Pushpa Narayanaswami et al.   NEUROLOGY

Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy

(2015) Nicolas Wein et al.   PEDIATRIC CLINICS OF NORTH AMERICA

Spinal Muscular Atrophies

(2015) Basil T. Darras   PEDIATRIC CLINICS OF NORTH AMERICA

Prevalence of Duchenne and Becker Muscular Dystrophies in the United States

(2015) P. A. Romitti et al.   PEDIATRICS

A comprehensive genomic approach for neuromuscular diseases gives a high diagnostic yield

(2014) Arunkanth Ankala et al.   ANNALS OF NEUROLOGY

Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

(2014) B Wu et al.   GENE THERAPY

Adult care for Duchenne muscular dystrophy in the UK

(2014) Sunil Rodger et al.   JOURNAL OF NEUROLOGY

Spinal muscular atrophy: Diagnosis and management in a new therapeutic era

(2014) W. David Arnold et al.   MUSCLE & NERVE

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

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

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

Whole-body vibration training in children with Duchenne muscular dystrophy and spinal muscular atrophy

(2013) Julia Vry et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

All-Cause Mortality and Cardiovascular Outcomes With Prophylactic Steroid Therapy in Duchenne Muscular Dystrophy

(2013) Gernot Schram et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

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(2013) Silvia Consalvi et al.   MOLECULAR MEDICINE

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

(2013) Michele A Scully et al.   Expert Opinion on Orphan Drugs

Respiratory management strategies for Duchenne muscular dystrophy: practice variation amongst canadian sub-specialists

(2012) Sherri L. Katz et al.   PEDIATRIC PULMONOLOGY

A Practical Approach to Molecular Diagnostic Testing in Neuromuscular Diseases

(2012) W. David Arnold et al.   Physical Medicine and Rehabilitation Clinics of North America

Exercise in Neuromuscular Diseases

(2012) R. Ted Abresch et al.   Physical Medicine and Rehabilitation Clinics of North America

Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72 400 specimens

(2011) Elaine A Sugarman et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

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

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

Duchenne Muscular Dystrophy: Continuous Noninvasive Ventilatory Support Prolongs Survival

(2011) J. R. Bach et al.   Respiratory Care

Serial Casting for the Management of Ankle Contracture in Duchenne Muscular Dystrophy

(2011) Allan M. Glanzman et al.   Pediatric Physical Therapy

Duchenne muscular dystrophy: Survival by cardio-respiratory interventions

(2010) Yuka Ishikawa et al.   NEUROMUSCULAR DISORDERS

Prevalence of genetic muscle disease in Northern England: in-depth analysis of a muscle clinic population

(2009) Fiona L. M. Norwood et al.   BRAIN

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

A novel custom high density-comparative genomic hybridization array detects common rearrangements as well as deep intronic mutations in dystrophinopathies

(2008) Matteo Bovolenta et al.   BMC GENOMICS

Microarray-based mutation detection in thedystrophingene

(2008) Madhuri R. Hegde et al.   HUMAN MUTATION

Neuropsychiatric Disorders in Males With Duchenne Muscular Dystrophy: Frequency Rate of Attention-Deficit Hyperactivity Disorder (ADHD), Autism Spectrum Disorder, and Obsessive—Compulsive Disorder

(2008) Joseph G. M. Hendriksen et al.   JOURNAL OF CHILD NEUROLOGY

HDAC2 blockade by nitric oxide and histone deacetylase inhibitors reveals a common target in Duchenne muscular dystrophy treatment

(2008) C. Colussi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA