Preclinical drug trials in the mdx mouse: assessment of reliable and sensitive outcome measures.


Journal Article

The availability of animal models for Duchenne muscular dystrophy has led to extensive preclinical research on potential therapeutics. Few studies have focused on reliability and sensitivity of endpoints for mdx mouse drug trials. Therefore, we sought to compare a wide variety of reported and novel endpoint measures in exercised mdx and normal control mice at 10, 20, and 40 weeks of age. Statistical analysis as well as power calculations for expected effect sizes in mdx preclinical drug trials across different ages showed that body weight, normalized grip strength, horizontal activity, rest time, cardiac function measurements, blood pressure, total central/peripheral nuclei per fiber, and serum creatine kinase are the most effective measurements for detecting drug-induced changes. These data provide an experimental basis upon which standardization of preclinical drug testing can be developed. Muscle Nerve, 2008.

Full Text

Duke Authors

Cited Authors

  • Spurney, CF; Gordish-Dressman, H; Guerron, AD; Sali, A; Pandey, GS; Rawat, R; Van Der Meulen, JH; Cha, H-J; Pistilli, EE; Partridge, TA; Hoffman, EP; Nagaraju, K

Published Date

  • May 2009

Published In

Volume / Issue

  • 39 / 5

Start / End Page

  • 591 - 602

PubMed ID

  • 19260102

Pubmed Central ID

  • 19260102

International Standard Serial Number (ISSN)

  • 0148-639X

Digital Object Identifier (DOI)

  • 10.1002/mus.21211


  • eng

Conference Location

  • United States