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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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T.I.3 Antisense Oligonucleotide (AON) mediated exon skipping trials in Duchenne Muscular Dystrophy (DMD): Current status, future prospects and challenges

Neuromuscular disorders : NMD, 2011-10, Vol.21 (9), p.702-702

Goemans, N

2011

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P 003 – Gait deviations in children with Charcot-Marie Tooth’s disease and their association with muscle weakness

Gait & posture, 2018-09, Vol.65, p.239-240

2018

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Development of a patient‐reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM

Developmental medicine and child neurology, 2017-02, Vol.59 (2), p.224-231

2017

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Myotonic dystrophy type 1: Long term natural history data

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S195-S196

2016

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P 148 – Synergy complexity during maximal voluntary isometric contractions

Gait & posture, 2018-09, Vol.65, p.480-481

2018

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P 148 - Synergy complexity during maximal voluntary isometric contractions

Gait & posture, 2018-09, Vol.65, p.203-204

2018

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Development of a prognostic model for 1-year change in 6-minute walk distance (6MWD) in patients with Duchenne muscular dystrophy (DMD)

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S182-S183

2016

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Comparing clinical phenotype of patients with Duchenne muscular dystrophy with deletions amenable to skipping exons 44, 45, and 53

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S95-S95

2016

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Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne

Scientific reports, 2017-12, Vol.7 (1), p.17888-10, Article 17888

2017

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Idebenone treatment in Friedreich's ataxia: neurological, cardiac, and biochemical monitoring

Neurology, 2003-05, Vol.60 (10), p.1679

2003

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Toward an appropriate neuropsychological assessment protocol for children with myotonic dystrophy type 1

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S196-S196

2016

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Normative data and reference equation for the six-minute walk test in healthy Caucasian boys aged 13–18 years

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S183-S183

2016

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Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients

Neuromuscular disorders : NMD, 2014-04, Vol.24 (4), p.347-352

2014

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T.P.6

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.862-862

2014

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A prospective natural history study to measure progression of physical impairment, activity limitation and quality of life in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S302-S302

2015

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Assessment of early motor development in young boys with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S228-S229

2015

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Longitudinal quantitative muscle MRI in 5 Duchenne boys on and off drisapersen treatment

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S198-S198

2015

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Longitudinal quantitative muscle magnetic resonance imaging (qMRI) in five boys with Duchenne muscular dystrophy (DMD), on and off treatment with drisapersen

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S198-S198

2015

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Reference values for the three-minute walk test, North Star ambulatory assessment and timed tests in typically developing boys aged 2.5–5 years

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S228-S228

2015

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Six minute walk test in type III spinal muscular atrophy: A 12 month longitudinal study

Neuromuscular disorders : NMD, 2013-08, Vol.23 (8), p.624-628

2013

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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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G.P.113

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.829-829

2014

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G.O.23

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.922-922

2014

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G.P.114

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.829-829

2014

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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

T.I.3 Antisense Oligonucleotide (AON) mediated exon skipping trials in Duchenne Muscular Dystrophy (DMD): Current status, future prospects and challenges

P 003 – Gait deviations in children with Charcot-Marie Tooth’s disease and their association with muscle weakness

Development of a patient‐reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM

Myotonic dystrophy type 1: Long term natural history data

P 148 – Synergy complexity during maximal voluntary isometric contractions

P 148 - Synergy complexity during maximal voluntary isometric contractions

Development of a prognostic model for 1-year change in 6-minute walk distance (6MWD) in patients with Duchenne muscular dystrophy (DMD)

Comparing clinical phenotype of patients with Duchenne muscular dystrophy with deletions amenable to skipping exons 44, 45, and 53

Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne

Idebenone treatment in Friedreich's ataxia: neurological, cardiac, and biochemical monitoring

Toward an appropriate neuropsychological assessment protocol for children with myotonic dystrophy type 1

Normative data and reference equation for the six-minute walk test in healthy Caucasian boys aged 13–18 years

Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients

T.P.6

A prospective natural history study to measure progression of physical impairment, activity limitation and quality of life in Duchenne muscular dystrophy

Assessment of early motor development in young boys with Duchenne muscular dystrophy

Longitudinal quantitative muscle MRI in 5 Duchenne boys on and off drisapersen treatment

Longitudinal quantitative muscle magnetic resonance imaging (qMRI) in five boys with Duchenne muscular dystrophy (DMD), on and off treatment with drisapersen

Reference values for the three-minute walk test, North Star ambulatory assessment and timed tests in typically developing boys aged 2.5–5 years

Six minute walk test in type III spinal muscular atrophy: A 12 month longitudinal study

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

G.P.113

G.O.23

G.P.114

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