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THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

Muscle & nerve, 2013-09, Vol.48 (3), p.343-356

2013

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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

The Lancet (British edition), 2015-05, Vol.385 (9979), p.1748-1757

2015

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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

Muscle & nerve, 2013-09, Vol.48 (3), p.357-368

2013

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Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials

PloS one, 2024-06, Vol.19 (6), p.e0304099-e0304099

2024

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Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study

Annals of neurology, 2015-04, Vol.77 (4), p.684-696

2015

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Clinical Approach to the Diagnostic Evaluation of Hereditary and Acquired Neuromuscular Diseases

Physical medicine and rehabilitation clinics of North America, 2012-08, Vol.23 (3), p.495-563

McDonald, Craig M., MD

2012

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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy

Muscle & nerve, 2010-04, Vol.41 (4), p.500-510

2010

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Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial

PloS one, 2022-08, Vol.17 (8), p.e0272858-e0272858

2022

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Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2020-06, Vol.30 (6), p.492-502

2020

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Gait Characterization in Duchenne Muscular Dystrophy (DMD) Using a Single-Sensor Accelerometer: Classical Machine Learning and Deep Learning Approaches

Sensors (Basel, Switzerland), 2024-02, Vol.24 (4), p.1123

2024

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Mexiletine for muscle cramps in amyotrophic lateral sclerosis: A randomized, double‐blind crossover trial

Muscle & nerve, 2018-07, Vol.58 (1), p.42-48

2018

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Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds: Automated Approach

Sensors (Basel, Switzerland), 2024-02, Vol.24 (4), p.1155

2024

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Prevention and Management of Limb Contractures in Neuromuscular Diseases

Physical medicine and rehabilitation clinics of North America, 2012-08, Vol.23 (3), p.675-687

2012

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Medical management of muscle weakness in Duchenne muscular dystrophy

PloS one, 2020-10, Vol.15 (10), p.e0240687-e0240687

2020

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First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

Digital biomarkers, 2021-05, Vol.5 (2), p.183-190

2021

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Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies

Journal of the American Society of Echocardiography, 2015-08, Vol.28 (8), p.999-1008

2015

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Sleep Disordered Breathing in Children with Neuromuscular Disease

Children (Basel), 2023-10, Vol.10 (10), p.1675

2023

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Reachable workspace and performance of upper limb (PUL) in duchenne muscular dystrophy

Muscle & nerve, 2016-04, Vol.53 (4), p.545-554

2016

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Influence of β2 adrenergic receptor genotype on risk of nocturnal ventilation in patients with Duchenne muscular dystrophy

Respiratory research, 2019-10, Vol.20 (1), p.221-221, Article 221

2019

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Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

Frontiers in cell and developmental biology, 2023-07, Vol.11, p.1167762-1167762

2023

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Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

JAMA neurology, 2020-08, Vol.77 (8), p.982

2020

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Physical activity, health impairments, and disability in neuromuscular disease

American journal of physical medicine & rehabilitation, 2002-11, Vol.81 (11 Suppl), p.S108-S120

McDonald, Craig M

2002

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Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

Journal of comparative effectiveness research, 2023-04, Vol.12 (4), p.e220190-e220190

2023

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Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

BMC cardiovascular disorders, 2022-06, Vol.22 (1), p.260-260, Article 260

2022

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One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development

Pediatric neurology, 2014-06, Vol.50 (6), p.557-563

2014

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THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

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

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials

Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study

Clinical Approach to the Diagnostic Evaluation of Hereditary and Acquired Neuromuscular Diseases

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy

Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial

Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

Gait Characterization in Duchenne Muscular Dystrophy (DMD) Using a Single-Sensor Accelerometer: Classical Machine Learning and Deep Learning Approaches

Mexiletine for muscle cramps in amyotrophic lateral sclerosis: A randomized, double‐blind crossover trial

Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds: Automated Approach

Prevention and Management of Limb Contractures in Neuromuscular Diseases

Medical management of muscle weakness in Duchenne muscular dystrophy

First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies

Sleep Disordered Breathing in Children with Neuromuscular Disease

Reachable workspace and performance of upper limb (PUL) in duchenne muscular dystrophy

Influence of β2 adrenergic receptor genotype on risk of nocturnal ventilation in patients with Duchenne muscular dystrophy

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

Physical activity, health impairments, and disability in neuromuscular disease

Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development

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