Jove
Visualize
联系我们
JoVE
x logofacebook logolinkedin logoyoutube logo
关于 JoVE
概览领导团队博客JoVE 帮助中心
作者
出版流程编辑委员会范围与政策同行评审常见问题投稿
图书馆员
用户评价订阅访问资源图书馆顾问委员会常见问题
研究
JoVE JournalMethods CollectionsJoVE Encyclopedia of Experiments存档
教育
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab Manual教师资源中心教师网站
使用条款与条件
隐私政策
政策

相关概念视频

Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

2.0K
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...
2.0K

您也可能阅读

相关文章

通过共同作者、期刊和引用图与本文相关的文章。

排序
Same author

Evaluating the Impact of Different Natural History Modeling Methods on Cost-Effectiveness Decisions: A Case Study in Duchenne Muscular Dystrophy.

MDM policy & practice·2026
Same author

Black Hole Spectroscopy and Tests of General Relativity with GW250114.

Physical review letters·2026
Same author

Patient and Public Involvement (PPI) in Secondary Data Analysis: Protocol for a Targeted Review.

Health expectations : an international journal of public participation in health care and health policy·2025
Same author

GW250114: Testing Hawking's Area Law and the Kerr Nature of Black Holes.

Physical review letters·2025
Same author

Liver function in X-linked myotubular myopathy and autosomal dominant centronuclear myopathy: Data of the unite-CNM study.

Journal of neuromuscular diseases·2025
Same author

Correction: Developing a Natural History Model for Duchenne Muscular Dystrophy.

PharmacoEconomics - open·2024

相关实验视频

Updated: Jul 9, 2025

Assessing Functional Performance in the Mdx Mouse Model
10:32

Assessing Functional Performance in the Mdx Mouse Model

Published on: March 27, 2014

32.6K

开发一个自然历史模型的杜恩肌肉发育不良症.

Jonathan Broomfield1, M Hill2, F Chandler3,4

  • 1University of Leicester, Leicester, UK. jb781@leicester.ac.uk.

PharmacoEconomics - open
|November 29, 2023
PubMed
概括

杜恩肌肉发育不良 (DMD) 的新自然史模型 (NHM) 估计了疾病的进展和存活率,有助于对新疗法的经济评估.

更多相关视频

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
09:18

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

Published on: January 12, 2019

10.4K
Isometric and Eccentric Force Generation Assessment of Skeletal Muscles Isolated from Murine Models of Muscular Dystrophies
14:10

Isometric and Eccentric Force Generation Assessment of Skeletal Muscles Isolated from Murine Models of Muscular Dystrophies

Published on: January 31, 2013

33.0K

相关实验视频

Last Updated: Jul 9, 2025

Assessing Functional Performance in the Mdx Mouse Model
10:32

Assessing Functional Performance in the Mdx Mouse Model

Published on: March 27, 2014

32.6K
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
09:18

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

Published on: January 12, 2019

10.4K
Isometric and Eccentric Force Generation Assessment of Skeletal Muscles Isolated from Murine Models of Muscular Dystrophies
14:10

Isometric and Eccentric Force Generation Assessment of Skeletal Muscles Isolated from Murine Models of Muscular Dystrophies

Published on: January 31, 2013

33.0K

科学领域:

  • 生物医学信息学 生物医学信息学
  • 疾病建模 疾病建模
  • 卫生经济学 卫生经济学

背景情况:

  • 杜恩肌肉发育不良症 (DMD) 需要强大的工具来进行医疗技术评估.
  • 现有的模型缺乏对疾病轨迹的全面,以患者为中心的观点.
  • 赫尔库勒斯项目旨在开发基于数据的模型,用于DMD治疗评估.

研究的目的:

  • 开发一个以患者为中心,以数据为基础的自然史模型 (NHM) 来治疗杜恩肌肉发育不良 (DMD).
  • 根据当前的护理标准,估计DMD患者的终身疾病轨迹.
  • 为了支持未来的经济评估和医疗技术评估,用于新的DMD疗法.

主要方法:

  • 聚合了多个数据集,包括关键路径研究所杜琴监管科学联盟 (D-RSC) 数据库.
  • 基于临床试验结果,现实实践和专家意见的定义健康状况.
  • 补充了数据与专家的诱导和系统的文献审查和死亡率的元分析.

主要成果:

  • 该NHM结合了健行,转移和非健行阶段,详细介绍了功能衰退.
  • 患者在门诊状态中花费大约9.5年,在转移状态中花费1.5年.
  • 据估计,DMD患者的中位预测存活时间为34.8年.

结论:

  • 开发的NHM为DMD提供了详细的疾病途径,包括关键转移状态.
  • 这种模型可以通过结合DMD的终身时间视界来增强经济评估.
  • 该NHM将有助于确定杜恩肌肉衰竭的未来研究重点.