Congenital muscular dystrophy: searching for a definition after 98 years

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice.

Gene therapy·2015

AAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy.

Gene therapy·2015

The 2-min walk test is sufficient for evaluating walking abilities in sporadic inclusion body myositis.

Neuromuscular disorders : NMD·2013

Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.

Molecular therapy : the journal of the American Society of Gene Therapy·2013

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

Neurology·2008

Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy.

Neurology·2007

Prevalence of Slowly Expanding Lesions in Patients With Multiple Sclerosis: A Systematic Review and Meta-Analysis.

Neurology·2026

Pearls & Oy-sters: DPPX Antibody-Associated Encephalitis in a Patient With Diarrhea, Tremor, Ocular Flutter, and Cognitive-Psychiatric Changes.

Neurology·2026

Clinical Reasoning: A Patient With Progressive Sensory Neuropathy: Using Nerve Ultrasound and Skin Biopsy to Refine Diagnostic Reasoning.

Neurology·2026

Longitudinal Dynamics of the Blood Proteome Within the Alzheimer Spectrum in Down Syndrome.

Neurology·2026

Pharmacologic Treatment for Migraine Prevention in Adults Practice Guideline Recommendations: Report of the AAN Guidelines Subcommittee and the American Headache Society.

Neurology·2026

Systematic Review of Pharmacologic Treatment for Migraine Prevention in Adults: Report of the AAN Guidelines Subcommittee and the American Headache Society.

Neurology·2026

Exploring Limits of [1-13C]Pyruvate Nuclear Spin Relaxation: Impact of Field, Deuteration, Degassing, and Additives.

Small science·2026

Using hyperpolarised xenon magnetic resonance imaging to explore breathlessness in long COVID: results from the EXPLAIN study.

ERJ open research·2026

Purification and Characterization of Extracellular Vesicles from Embryo-Conditioned Medium.

Methods in molecular biology (Clifton, N.J.)·2026

Low-MESF fluorescent reference particles for flow cytometry analysis of extracellular vesicles.

Scientific reports·2026

Quantitative classification of asbestos-like morphology for assessing potentially hazardous fibrous minerals.

Journal of hazardous materials·2026

Cofilin 1 Is an Extracellular Vesicle-Associated Plasma Biomarker of Parkinson's Disease in Humans.

Annals of neurology·2026

Related Experiment Videos

Congenital muscular dystrophy: searching for a definition after 98 years

Related Concept Videos

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice.

AAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy.

The 2-min walk test is sufficient for evaluating walking abilities in sporadic inclusion body myositis.

Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy.

Prevalence of Slowly Expanding Lesions in Patients With Multiple Sclerosis: A Systematic Review and Meta-Analysis.

Pearls & Oy-sters: DPPX Antibody-Associated Encephalitis in a Patient With Diarrhea, Tremor, Ocular Flutter, and Cognitive-Psychiatric Changes.

Clinical Reasoning: A Patient With Progressive Sensory Neuropathy: Using Nerve Ultrasound and Skin Biopsy to Refine Diagnostic Reasoning.

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Pharmacologic Treatment for Migraine Prevention in Adults Practice Guideline Recommendations: Report of the AAN Guidelines Subcommittee and the American Headache Society.

Systematic Review of Pharmacologic Treatment for Migraine Prevention in Adults: Report of the AAN Guidelines Subcommittee and the American Headache Society.

Exploring Limits of [1-13C]Pyruvate Nuclear Spin Relaxation: Impact of Field, Deuteration, Degassing, and Additives.

Using hyperpolarised xenon magnetic resonance imaging to explore breathlessness in long COVID: results from the EXPLAIN study.

Purification and Characterization of Extracellular Vesicles from Embryo-Conditioned Medium.

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Congenital muscular dystrophy: searching for a definition after 98 years

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