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Preclinical drug trials in the mdx mouse: Assessment of reliable and sensitive outcome measures
Author(s) -
Spurney Christopher F.,
GordishDressman Heather,
Guerron Alfredo D.,
Sali Arpana,
Pandey Gouri S.,
Rawat Rashmi,
Van Der Meulen Jack H.,
Cha HeeJae,
Pistilli Emidio E.,
Partridge Terence A.,
Hoffman Eric P.,
Nagaraju Kanneboyina
Publication year - 2009
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.21211
Subject(s) - duchenne muscular dystrophy , mdx mouse , medicine , drug , muscular dystrophy , pharmacology , grip strength , clinical trial , physical medicine and rehabilitation , oncology , physical therapy , dystrophin
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