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Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

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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Assessing Functional Performance in the Mdx Mouse Model
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Published on: March 27, 2014

A model-informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy.

Jongjin Kim1,2, Juan Francisco Morales1, Sanghoon Kang1

  • 1Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA.

CPT: Pharmacometrics & Systems Pharmacology
|October 3, 2024
PubMed
Summary

This tutorial guides developers in creating R Shiny apps for model-based clinical trial simulations. It demonstrates building a Duchenne muscular dystrophy simulation tool, enhancing efficiency and reducing participant burden in drug development.

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Last Updated: Jun 14, 2026

Assessing Functional Performance in the Mdx Mouse Model
10:32

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Published on: March 27, 2014

A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy
05:54

A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy

Published on: December 29, 2017

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

Area of Science:

  • Biostatistics
  • Computational Biology
  • Drug Development

Background:

  • Model-based clinical trial simulation tools are essential for optimizing study designs.
  • Developing user-friendly simulation platforms requires expertise in both modeling and interface development.
  • Existing tools often lack the interactive features needed for efficient scenario exploration.

Purpose of the Study:

  • To provide a tutorial for developing tailored R Shiny apps for model-based clinical trial simulation.
  • To demonstrate the creation of a specific simulation tool for Duchenne muscular dystrophy (DMD).
  • To guide developers in building interactive platforms for exploring clinical trial designs.

Main Methods:

  • Development of an R Shiny application for clinical trial simulation.
  • Creation of a virtual population using machine learning to augment sample size.
  • Implementation of structural framework, controllers, and visualization techniques within the app.
  • External validation of simulation outputs against a real-world clinical trial placebo arm.

Main Results:

  • A functional R Shiny app for model-based clinical trial simulation was successfully developed.
  • The tool incorporates key features like criteria selection and power calculation.
  • Virtual population generation enhanced the simulation's capacity.
  • External validation confirmed the reliability of the simulated outputs.

Conclusions:

  • The tutorial provides a practical framework for developing interactive clinical trial simulation tools.
  • The presented methods are applicable to Duchenne muscular dystrophy progression models and can be adapted for other diseases.
  • This approach can improve the efficiency of clinical trial design and reduce participant burden.