Related references
Note: Only part of the references are listed.Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy
Anne M. Connolly et al.
MUSCLE & NERVE (2019)
Pilot study of population-based newborn screening for spinal muscular atrophy in New York state
Jennifer N. Kraszewski et al.
GENETICS IN MEDICINE (2018)
Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan
David J. Birnkrant et al.
LANCET NEUROLOGY (2018)
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management (vol 17, pg 251, 2018)
J. F. Zimmermann et al.
LANCET NEUROLOGY (2018)
Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management
David J. Birnkrant et al.
LANCET NEUROLOGY (2018)
Why patients prefer high-level healthcare facilities: a qualitative study using focus groups in rural and urban China
Yun Liu et al.
BMJ GLOBAL HEALTH (2018)
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
David J. Birnkrant et al.
LANCET NEUROLOGY (2018)
Advances in spinal muscular atrophy therapeutics
Valeria Parente et al.
THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS (2018)
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Craig M. McDonald et al.
LANCET (2017)
Clinical and mutational characteristics of Duchenne muscular dystrophy patients based on a comprehensive database in South China
Dan-Ni Wang et al.
NEUROMUSCULAR DISORDERS (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
R. S. Finkel et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Newborn screening for Duchenne muscular dystrophy in China: follow-up diagnosis and subsequent treatment
Qing Ke et al.
WORLD JOURNAL OF PEDIATRICS (2017)
ISS-N1 MAKES THE FIRST FDA-APPROVED DRUG FOR SPINAL MUSCULAR ATROPHY
Eric W. Ottesen
TRANSLATIONAL NEUROSCIENCE (2017)
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela Guglieri et al.
CONTEMPORARY CLINICAL TRIALS (2017)
TWENTY-YEAR FOLLOW-UP OF NEWBORN SCREENING FOR PATIENTS WITH MUSCULAR DYSTROPHY
Jeffrey Chung et al.
MUSCLE & NERVE (2016)
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2016)
CLINICAL TRIAL READINESS IN NON-AMBULATORY BOYS AND MEN WITH DUCHENNE MUSCULAR DYSTROPHY: MDA-DMD NETWORK FOLLOW-UP
Anne M. Connolly et al.
MUSCLE & NERVE (2016)
CLINICAL FOLLOW-UP FOR DUCHENNE MUSCULAR DYSTROPHY NEWBORN SCREENING: A PROPOSAL
Jennifer M. Kwon et al.
MUSCLE & NERVE (2016)
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy
Robert C. Griggs et al.
NEUROLOGY (2016)
Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy Report of the Guideline Development Subcommittee of the American Academy of Neurology
David Gloss et al.
NEUROLOGY (2016)
OUTCOME RELIABILITY IN NON-AMBULATORY BOYS/MEN WITH DUCHENNE MUSCULAR DYSTROPHY
Anne M. Connolly et al.
MUSCLE & NERVE (2015)
Delay in Diagnosis of Spinal Muscular Atrophy: A Systematic Literature Review
Chia-Wei Lin et al.
PEDIATRIC NEUROLOGY (2015)
ATALUREN TREATMENT OF PATIENTS WITH NONSENSE MUTATION DYSTROPHINOPATHY
Katharine Bushby et al.
MUSCLE & NERVE (2014)
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2013)
Corticosteroids in Duchenne muscular dystrophy: Major variations in practice
Robert C. Griggs et al.
MUSCLE & NERVE (2013)
Survival of Patients With Spinal Muscular Atrophy Type 1
Cesare Gregoretti et al.
PEDIATRICS (2013)
Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2012)
Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of > 72 400 specimens
Elaine A. Sugarman et al.
EUROPEAN JOURNAL OF HUMAN GENETICS (2012)
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
Elisa Dominguez et al.
HUMAN MOLECULAR GENETICS (2011)
Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy
D. M. Escolar et al.
NEUROLOGY (2011)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
Chiara F. Valori et al.
SCIENCE TRANSLATIONAL MEDICINE (2010)
Delayed Diagnosis in Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)
Emma Ciafaloni et al.
JOURNAL OF PEDIATRICS (2009)
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. (vol 8, pg 918, 2009)
M. Kinali et al.
LANCET NEUROLOGY (2009)
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
Maria Kinali et al.
LANCET NEUROLOGY (2009)
Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle
Maja Z. Salva et al.
MOLECULAR THERAPY (2007)
Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort
KM Dent et al.
AMERICAN JOURNAL OF MEDICAL GENETICS PART A (2005)
High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy
AM Connolly et al.
NEUROMUSCULAR DISORDERS (2002)
Share Paper
