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相关概念视频

Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

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Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
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Disorders of the Skeletal Muscle01:28

Disorders of the Skeletal Muscle

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The clinical conditions affecting the skeletal muscle tissue are broadly categorized as musculoskeletal and neuromuscular disorders.
Musculoskeletal disorders
Musculoskeletal disorders involve injuries and conditions affecting the skeletal muscles and associated connective tissues. These disorders can arise from acute biomechanical stresses or chronic overuse and can occur across different age groups. Common injuries include sprains, fractures, and muscular strains, often resulting from...
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Myasthenia Gravis: Overview and Treatment01:20

Myasthenia Gravis: Overview and Treatment

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Myasthenia gravis is a neuromuscular transmission disorder characterized by weakness and increased fatigability of skeletal muscles. It is an autoimmune disease affecting approximately one in 2000 people, where antibodies against the α1 subunit of nicotinic acetylcholine receptors are produced.
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which...
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相关实验视频

Updated: Jul 19, 2025

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

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关于贝克尔肌肉发育不良症的最新情况

Volker Straub1, Michela Guglieri

  • 1John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Current opinion in neurology
|August 17, 2023
PubMed
概括

最近的研究进展 贝克尔肌肉缩症 (BMD) 试验准备和自然历史研究. 尽管取得了进展,但长期数据和疾病变异性挑战了这种神经肌肉疾病的临床试验设计.

科学领域:

  • 神经学 神经学
  • 遗传学 是一个遗传学.
  • 临床研究 临床研究

背景情况:

  • 贝克尔肌肉发育不良 (BMD) 正在获得研究重点,部分原因是旨在将杜申肌肉发育不良 (DMD) 现型转移到BMD的治疗策略.
  • 诊断和临床前模型的进步正在支持BMD的研究.

结论:

  • 尽管取得了进展,但仍然缺乏BMD的长期自然史数据.
  • 在BMD中疾病严重程度的广泛范围对设计有效的临床试验提出了重大挑战.

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Last Updated: Jul 19, 2025

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