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155P The experience of parents as they make treatment decisions for their child with spinal muscular atrophy and the factors that influence these decisions: a narrative review

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.590

2024

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Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

Journal of pharmacokinetics and pharmacodynamics, 2019-10, Vol.46 (5), p.441-455

2019

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CAT-1004, an oral agent targeting NF-kB: MoveDMD trial results in Duchenne muscular dystrophy (DMD)

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S157-S157

2016

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Genetic polymorphisms modify intramuscular fat infiltration in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S254-S255

2015

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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

The New England journal of medicine, 2017-11, Vol.377 (18), p.1723-1732

2017

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394P Longitudinal changes in the North Star Ambulatory Assessment and leg muscle fat fraction in DMD

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.122

2024

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409P Short-term and long-term safety profile of Givinostat in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2024-10, Vol.43, p.104441, Article 104441.280

2024

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P.101 Baseline characteristics/initial safety in RESPOND: phase 4 study of nusinersen in children with SMA who previously received onasemnogene abeparvovec

Neuromuscular disorders : NMD, 2022-10, Vol.32, p.S86

2022

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P.69 Minimum clinically important difference in magnetic resonance biomarkers in DMD

Neuromuscular disorders : NMD, 2022-10, Vol.32, p.S69-S70

2022

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DMD – THERAPY

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S129

2020

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DMD – THERAPY: P.282 Edasalonexent treatment in young boys with Duchenne muscular dystrophy is associated with age-normative growth and normal adrenal function

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S129-S129

2020

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DUCHENNE MUSCULAR DYSTROPHY – IMAGING AND BIOMARKERS

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S41

2018

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DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY: P.305Disease progression in arm versus leg muscles in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S123-S123

2018

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DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S123

2018

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DUCHENNE MUSCULAR DYSTROPHY – IMAGING AND BIOMARKERS: P.34MR biomarkers in imaging DMD clinical trial network

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S41-S41

2018

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O.42Treatment of young boys with Duchenne muscular dystrophy with the NF-κB inhibitor edasalonexent showed a slowing of disease progression as assessed by MRI and functional measures

Neuromuscular disorders : NMD, 2019-10, Vol.29, p.S208-S208

2019

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MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S215

2017

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P.397 - MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S215-S215

2017

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DMD CLINICAL THERAPIES I: P.116Edasalonexent, an NF-κB Inhibitor, slows disease progression over more than a year compared to control period in 4 to 7 year old patients with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S64-S64

2018

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DMD CLINICAL THERAPIES I

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S64

2018

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P.230 - CAT-1004, an oral agent targeting NF-kB: MoveDMD trial results in Duchenne muscular dystrophy (DMD)

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S157-S157

2016

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Magnetic resonance biomarkers in the proximal and distal upper extremity in a large cohort of boys with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S125

2017

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P.92 - Magnetic resonance biomarkers in the proximal and distal upper extremity in a large cohort of boys with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S125-S125

2017

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G.P.227 - Genetic polymorphisms modify intramuscular fat infiltration in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S254-S255

2015

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Lack of association between temporal lobe epilepsy and a novel polymorphism in the alpha 2 subunit gene (ATP1A2) of the sodium potassium transporting ATPase

American journal of medical genetics, 2000-02, Vol.96 (1), p.79-83

2000

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155P The experience of parents as they make treatment decisions for their child with spinal muscular atrophy and the factors that influence these decisions: a narrative review

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

CAT-1004, an oral agent targeting NF-kB: MoveDMD trial results in Duchenne muscular dystrophy (DMD)

Genetic polymorphisms modify intramuscular fat infiltration in Duchenne muscular dystrophy

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

394P Longitudinal changes in the North Star Ambulatory Assessment and leg muscle fat fraction in DMD

409P Short-term and long-term safety profile of Givinostat in Duchenne muscular dystrophy

P.101 Baseline characteristics/initial safety in RESPOND: phase 4 study of nusinersen in children with SMA who previously received onasemnogene abeparvovec

P.69 Minimum clinically important difference in magnetic resonance biomarkers in DMD

DMD – THERAPY

DMD – THERAPY: P.282 Edasalonexent treatment in young boys with Duchenne muscular dystrophy is associated with age-normative growth and normal adrenal function

DUCHENNE MUSCULAR DYSTROPHY – IMAGING AND BIOMARKERS

DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY: P.305Disease progression in arm versus leg muscles in Duchenne muscular dystrophy

DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

DUCHENNE MUSCULAR DYSTROPHY – IMAGING AND BIOMARKERS: P.34MR biomarkers in imaging DMD clinical trial network

O.42Treatment of young boys with Duchenne muscular dystrophy with the NF-κB inhibitor edasalonexent showed a slowing of disease progression as assessed by MRI and functional measures

MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dystrophy

P.397 - MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dystrophy

DMD CLINICAL THERAPIES I: P.116Edasalonexent, an NF-κB Inhibitor, slows disease progression over more than a year compared to control period in 4 to 7 year old patients with Duchenne muscular dystrophy

DMD CLINICAL THERAPIES I

P.230 - CAT-1004, an oral agent targeting NF-kB: MoveDMD trial results in Duchenne muscular dystrophy (DMD)

Magnetic resonance biomarkers in the proximal and distal upper extremity in a large cohort of boys with Duchenne muscular dystrophy

P.92 - Magnetic resonance biomarkers in the proximal and distal upper extremity in a large cohort of boys with Duchenne muscular dystrophy

G.P.227 - Genetic polymorphisms modify intramuscular fat infiltration in Duchenne muscular dystrophy

Lack of association between temporal lobe epilepsy and a novel polymorphism in the alpha 2 subunit gene (ATP1A2) of the sodium potassium transporting ATPase

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