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Motor neuron synapses with muscle fiber via electrical impulse transmission and neurotransmitter release, forming neuromuscular junctions , motor neuron, neuroscience
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SOD1-ALS from Gene Discovery to Targeted Therapeutics: A Comprehensive Review

Joshua Newman, Phillipe Corcia, Angela Genge

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder of upper and lower motor neurons that results in progressive motor impairment. ALS is the most common disease of motor neurons with an annual incidence of approximately 1.7–2.5 per 100,000 people. It is a terminal condition with a typical life expectancy of 2–5 years from symptom onset. […]

touchREVIEWS in Neurology. 2025;21(1):1–5:Online ahead of journal publication

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Updates in the Use of Vamorolone and Steroids in the Treatment of Duchenne Muscular Dystrophy

Bridget McGowan, Nancy L Kuntz

Duchenne muscular dystrophy (DMD) is an X-linked recessive, progressive and universally fatal disease in the spectrum of dystrophinopathies,1 with an incidence of 21.4 patients in 100,000 live male births worldwide.2,3 Historically, patients with DMD would lose ambulation by the age of 10 due ...

touchREVIEWS in Neurology. 2023;19(2):7-9
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Descriptive Characteristics of Males with Duchenne Muscular Dystrophy in Those Covered by Government (Medicaid) and Non-government (Private) Health Plans

Duchenne muscular dystrophy (DMD) is a progressive, X-linked recessive neuromuscular disorder, characterized by muscle weakness, wasting, and degeneration starting in early childhood.1,2 DMD is caused by a mutation in the dystrophin gene that leads to an absence of functional dystrophin ...

US Neurology. 2018;14(2):88–93 DOI: https://doi.org/10.17925/USN.2018.14.2.88
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Early Diagnosis and Treatment – The Use of Ataluren in the Effective Management of Duchenne Muscular Dystrophy

These are exciting times in Duchenne muscular dystrophy (DMD); the first disease-modifying drug for this indication has been approved in the European Union for ambulatory DMD in patients >5 years; other drug treatments are in the pipeline.1–5 These treatments can ...

European Neurological Review. 2018;13(1):31–7 DOI: https://doi.org/10.17925/ENR.2018.13.1.31
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Foreword - US Neurology, 2016;12(1):

Ruben I Kuzniecky

Welcome to the latest edition of US Neurology, which features a wide range of topical articles covering numerous areas of neurology that are of interest to the wider biomedical community. In this edition, we discuss treatment of Duchene Muscular Dystrophy (...
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Corticosteroids in Duchenne Muscular Dystrophy—A Deflazacort Review

Duchenne muscular dystrophy (DMD) is an X-linked disorder affecting one in 5,000 live male births which makes it the most common and most severe form of muscular dystrophy.1 The absence of the protein dystrophin leads to symptom onset typically between the ...

US Neurology, 2016;12(1):12–6 DOI: http://doi.org/10.17925/USN.2016.12.01.12

Duchenne Muscular Dystrophy and its Treatment Options

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New Perspectives in the Management of Duchenne Muscular Dystrophy

Report based on Presentations at a Satellite Symposium held at the European Paediatric Neurology Society (EPNS) on 25 September 2013 in Brussels, Belgium At the European Paediatric Neurology Society (EPNS) conference held in Brussels during September 2013, a satellite symposium sponsored by PTC ...

European Neurological Review, 2014;9(1):78–82 DOI: http://doi.org/10.17925/ENR.2014.09.01.78

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