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Loss of sarcolemmal nNOS is common in acquired and inherited neuromuscular disorders

Neurology, 2011-03, Vol.76 (11), p.960-967

2011

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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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Loss of sarcolemmal nNOS is common in acquired and inherited neuromuscular disorders

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

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