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Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

PloS one, 2016-09, Vol.11 (9), p.e0161955-e0161955

2016

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Duchenne muscular dystrophy

Nature reviews. Disease primers, 2021-02, Vol.7 (1), p.13-13, Article 13

2021

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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

The New England journal of medicine, 2011-04, Vol.364 (16), p.1513-1522

2011

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

Annals of neurology, 2016-02, Vol.79 (2), p.257-271

2016

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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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Gene therapy for spinal muscular atrophy: hope and caution

Lancet neurology, 2021-04, Vol.20 (4), p.251-252

Goemans, Nathalie

2021

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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

The New England journal of medicine, 2007-12, Vol.357 (26), p.2677-2686

2007

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Muscle weakness has a limited effect on motor control of gait in Duchenne muscular dystrophy

PloS one, 2020-09, Vol.15 (9), p.e0238445-e0238445

2020

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A new strength assessment to evaluate the association between muscle weakness and gait pathology in children with cerebral palsy

PloS one, 2018-01, Vol.13 (1), p.e0191097-e0191097

2018

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Novel defects in collagen XII and VI expand the mixed myopathy/Ehlers-Danlos syndrome spectrum and lead to variant-specific alterations in the extracellular matrix

Genetics in medicine, 2020-01, Vol.22 (1), p.112-123

2020

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Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration

PloS one, 2020-06, Vol.15 (6), p.e0232870-e0232870

2020

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Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy

PloS one, 2016-10, Vol.11 (10), p.e0164684-e0164684

2016

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Illness Perceptions and Illness Identity in Adolescents and Emerging Adults With Neuromuscular Disorders

Clinical child psychology and psychiatry, 2023-07, Vol.28 (3), p.983-996

2023

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How glucocorticoids change life in Duchenne muscular dystrophy

The Lancet (British edition), 2018-02, Vol.391 (10119), p.406-407

Goemans, Nathalie

2018

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Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular Dystrophy

Frontiers in human neuroscience, 2022-06, Vol.16

2022

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Six-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years

PloS one, 2013, Vol.8 (12), p.e84120-e84120

2013

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Instrumented strength assessment in typically developing children and children with a neural or neuromuscular disorder: A reliability, validity and responsiveness study

Frontiers in physiology, 2022-10, Vol.13, p.855222-855222

2022

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Gait classification for growing children with Duchenne muscular dystrophy

Scientific reports, 2024-05, Vol.14 (1), p.10828-10828

2024

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Comparison of two corticosteroid regimens on brain volumetrics in patients with Duchenne muscular dystrophy

Annals of clinical and translational neurology, 2023-12, Vol.10 (12), p.2324-2333

2023

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Dystrophin deficiency leads to dysfunctional glutamate clearance in iPSC derived astrocytes

Translational psychiatry, 2019-08, Vol.9 (1), p.200-21, Article 200

2019

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Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

Duchenne muscular dystrophy

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

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

Gene therapy for spinal muscular atrophy: hope and caution

Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

Muscle weakness has a limited effect on motor control of gait in Duchenne muscular dystrophy

A new strength assessment to evaluate the association between muscle weakness and gait pathology in children with cerebral palsy

Novel defects in collagen XII and VI expand the mixed myopathy/Ehlers-Danlos syndrome spectrum and lead to variant-specific alterations in the extracellular matrix

Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration

Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

Illness Perceptions and Illness Identity in Adolescents and Emerging Adults With Neuromuscular Disorders

How glucocorticoids change life in Duchenne muscular dystrophy

Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular Dystrophy

Six-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years

North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

Instrumented strength assessment in typically developing children and children with a neural or neuromuscular disorder: A reliability, validity and responsiveness study

Infantile spinal muscular atrophy with respiratory distress type 1 (SMARD1)

Gait classification for growing children with Duchenne muscular dystrophy

Comparison of two corticosteroid regimens on brain volumetrics in patients with Duchenne muscular dystrophy

Dystrophin deficiency leads to dysfunctional glutamate clearance in iPSC derived astrocytes

Correction: Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

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