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Evaluation of MMX1902 as an Oral Treatment for Duchenne Muscular Dystrophy

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Technical Report,15 Sep 2016,14 Sep 2017

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University of Southern California Los Angeles United States

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In the cardiac-focused study conducted, 10-week-old mdx mice were exercised at 15 mmin for 60 minutes 2 times a week for 10 weeks. Four different SQ treatment groups n 9group were evaluated wild-type controls and three groups of mdx mice vehicle saline and two MMX1902 doses 1.0, and 2.0 mgkgday. DMD-associated cardiomyopathy is a dilated cardiomyopathy and, as such, is marked by increased left ventricular volume and decreased ejection fraction with compensatory tachycardia. Following treatment and exercise, echocardiography showed daily MMX1902 treatment at the 2 mgkgday dose to reduce left ventricular end systolic volume, increase ejection fraction, and ameliorate tachycardia resulting in cardiac functional measures comparable to exercised wild-type control mice. Further, embryonic myosin heavy chain eMHC staining of the diaphragm showed a significant increase in eMHC positive muscle fibers with MMX1902 treatment, at both doses, supporting to the potential for MMX1902 treatment to stimulate and sustain regeneration even in the face of long-term, intensive exercise.

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  • Medicine and Medical Research
  • Anatomy and Physiology
  • Pharmacology

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