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Quantifying the burden of caregiving in Duchenne muscular dystrophy

Journal of neurology, 2016-05, Vol.263 (5), p.906-915

2016

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Short stature and pubertal delay in Duchenne muscular dystrophy

Archives of Disease in Childhood, 2016-01, Vol.101 (1), p.101-106

2016

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Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy

PloS one, 2013-11, Vol.8 (11), p.e80263-e80263

2013

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Cardiac involvement in female carriers of duchenne or becker muscular dystrophy

Muscle & nerve, 2017-06, Vol.55 (6), p.810-818

2017

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Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy

Scientific reports, 2022-11, Vol.12 (1), p.18762-18762, Article 18762

2022

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Editorial: Lessons Learned from Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation

Frontiers in genetics, 2022-07, Vol.13, p.840074-840074

2022

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Natural history of Duchenne muscular dystrophy in the United Kingdom: A descriptive study using the Clinical Practice Research Datalink

Brain and behavior, 2023-12, Vol.13 (12), p.e3331-n/a

2023

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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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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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Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy

Annals of clinical and translational neurology, 2023-12, Vol.10 (12), p.2285-2296

2023

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Decoding the transcriptome of Duchenne muscular dystrophy to the single nuclei level reveals clinical-genetic correlations

Cell death & disease, 2023-09, Vol.14 (9), p.596-596, Article 596

2023

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Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry

Journal of neurology, 2016-07, Vol.263 (7), p.1401-1408

2016

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The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

Acta neuropathologica communications, 2021-01, Vol.9 (1), p.7-7, Article 7

2021

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Recessive mutations in RYR1 are a common cause of congenital fiber type disproportion

Human mutation, 2010-07, Vol.31 (7), p.E1544-E1550

2010

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Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy

Journal of neuromuscular diseases, , Vol.2 (3), p.241-255

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Wild-type bone marrow cells ameliorate the phenotype of SOD1-G93A ALS mice and contribute to CNS, heart and skeletal muscle tissues

Brain (London, England : 1878), 2004-11, Vol.127 (11), p.2518-2532

2004

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Cardiac care of children with dystrophinopathy and females carrying DMD-gene variations

Open heart, 2022-10, Vol.9 (2), p.e001977

2022

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Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?

Endocrine Connections, 2023-09, Vol.12 (12), p.1-12

2023

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Fibronectin is a serum biomarker for Duchenne muscular dystrophy

Proteomics. Clinical applications, 2014-04, Vol.8 (3-4), p.269-278

2014

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

Quantifying the burden of caregiving in Duchenne muscular dystrophy

Short stature and pubertal delay in Duchenne muscular dystrophy

Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Cardiac involvement in female carriers of duchenne or becker muscular dystrophy

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy

Editorial: Lessons Learned from Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation

Natural history of Duchenne muscular dystrophy in the United Kingdom: A descriptive study using the Clinical Practice Research Datalink

Randomized phase 2 trial and open-label extension of domagrozumab 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

Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy

Imaging mass cytometry analysis of Becker muscular dystrophy muscle samples reveals different stages of muscle degeneration

Decoding the transcriptome of Duchenne muscular dystrophy to the single nuclei level reveals clinical-genetic correlations

Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry

The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

Recessive mutations in RYR1 are a common cause of congenital fiber type disproportion

Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy

Wild-type bone marrow cells ameliorate the phenotype of SOD1-G93A ALS mice and contribute to CNS, heart and skeletal muscle tissues

Cardiac care of children with dystrophinopathy and females carrying DMD-gene variations

Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?

Clinical, molecular, and protein correlations in a large sample of genetically diagnosed Italian limb girdle muscular dystrophy patients

Fibronectin is a serum biomarker for Duchenne muscular dystrophy

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