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Spatial transcriptomics reveal markers of histopathological changes in Duchenne muscular dystrophy mouse models

Nature communications, 2023-08, Vol.14 (1), p.4909-4909, Article 4909

2023

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Quantitative MRI and strength measurements in the assessment of muscle quality in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2014-05, Vol.24 (5), p.409-416

2014

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Natural disease history of mouse models for limb girdle muscular dystrophy types 2D and 2F

PloS one, 2017-08, Vol.12 (8), p.e0182704-e0182704

2017

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Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

Human gene therapy, 2007-09, Vol.18 (9), p.798-810

2007

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Natural disease history of the dy2J mouse model of laminin α2 (merosin)-deficient congenital muscular dystrophy

PloS one, 2018, Vol.13 (5), p.e0197388-e0197388

2018

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Neuro-Cardio-Autonomic Modulations in Children with Duchenne Muscular Dystrophy

Journal of neuromuscular diseases, 2023-01, Vol.10 (2), p.227-238

2023

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Integrated lipidomic and transcriptomic analyses identify altered nerve triglycerides in mouse models of prediabetes and type 2 diabetes

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Deacetylation Inhibition Reverses PABPN1-Dependent Muscle Wasting

iScience, 2019-02, Vol.12, p.318-332

2019

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Serum matrix metalloproteinase-9 (MMP-9) as a biomarker for monitoring disease progression in Duchenne muscular dystrophy (DMD)

Neuromuscular disorders : NMD, 2011-08, Vol.21 (8), p.569-578

2011

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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Altered in vitro muscle differentiation in X-linked myopathy with excessive autophagy

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy

Journal of neuromuscular diseases, 2021-01, Vol.8 (5), p.845-863

2021

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Interactions among ryanodine receptor isotypes contribute to muscle fiber type development and function

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Modelling the pathogenesis of X-linked distal hereditary motor neuropathy using patient-derived iPSCs

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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A comparison of the bone and growth phenotype of mdx , mdx:Cmah -/- and mdx:Utrn +/- murine models with the C57BL/10 wild-type mouse

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Bioinformatics and mutations leading to exon skipping

Methods in molecular biology (Clifton, N.J.), 2012-01, Vol.867, p.17

2012

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Optimizing RNA/ENA chimeric antisense oligonucleotides using in vitro splicing

Methods in molecular biology (Clifton, N.J.), 2012, Vol.867, p.131

2012

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Profiling Serum Antibodies Against Muscle Antigens in Facioscapulohumeral Muscular Dystrophy Finds No Disease-Specific Autoantibodies

Journal of neuromuscular diseases, 2021-01, Vol.8 (5), p.801-814

2021

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To dystrophin and beyond: an interview with Louis Kunkel

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Plasma Creatinine Level Does Not Predict Respiratory Function in Amyotrophic Lateral Sclerosis

Journal of neuromuscular diseases, 2021-01, Vol.8 (5), p.795-799

2021

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At the heart of genetic disease: an interview with Elizabeth McNally

Disease models & mechanisms, 2020-02, Vol.13 (2)

2020

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Rebuttal to: Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy, Verhaart et al. 2020

Journal of neuromuscular diseases, 2021-01, Vol.8 (5), p.865-866

2021

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Sharing “Negative” Results in Neuromuscular Research: A Positive Experience

Journal of neuromuscular diseases, 2021-01, Vol.8 (5), p.765-767

2021

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Association of HLA Class II Alleles with Disease Severity and Treatment Response in Iranian Patients with Myasthenia Gravis

Journal of neuromuscular diseases, 2021-01, Vol.8 (5), p.827-829

2021

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Spatial transcriptomics reveal markers of histopathological changes in Duchenne muscular dystrophy mouse models

Quantitative MRI and strength measurements in the assessment of muscle quality in Duchenne muscular dystrophy

Natural disease history of mouse models for limb girdle muscular dystrophy types 2D and 2F

Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

Natural disease history of the dy2J mouse model of laminin α2 (merosin)-deficient congenital muscular dystrophy

Neuro-Cardio-Autonomic Modulations in Children with Duchenne Muscular Dystrophy

Integrated lipidomic and transcriptomic analyses identify altered nerve triglycerides in mouse models of prediabetes and type 2 diabetes

Deacetylation Inhibition Reverses PABPN1-Dependent Muscle Wasting

Serum matrix metalloproteinase-9 (MMP-9) as a biomarker for monitoring disease progression in Duchenne muscular dystrophy (DMD)

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

Altered in vitro muscle differentiation in X-linked myopathy with excessive autophagy

Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy

Interactions among ryanodine receptor isotypes contribute to muscle fiber type development and function

Modelling the pathogenesis of X-linked distal hereditary motor neuropathy using patient-derived iPSCs

A comparison of the bone and growth phenotype of mdx , mdx:Cmah -/- and mdx:Utrn +/- murine models with the C57BL/10 wild-type mouse

Bioinformatics and mutations leading to exon skipping

Optimizing RNA/ENA chimeric antisense oligonucleotides using in vitro splicing

Profiling Serum Antibodies Against Muscle Antigens in Facioscapulohumeral Muscular Dystrophy Finds No Disease-Specific Autoantibodies

To dystrophin and beyond: an interview with Louis Kunkel

Plasma Creatinine Level Does Not Predict Respiratory Function in Amyotrophic Lateral Sclerosis

At the heart of genetic disease: an interview with Elizabeth McNally

Rebuttal to: Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy, Verhaart et al. 2020

Sharing “Negative” Results in Neuromuscular Research: A Positive Experience

Association of HLA Class II Alleles with Disease Severity and Treatment Response in Iranian Patients with Myasthenia Gravis

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