Muscular Dystrophy News Forums › Forums › Diagnosis​ ​Info​ ​and​ ​General​ ​Questions › New Compound Might Limit Muscle Damage in MD Type 1

  • Diagnosis​ ​Info​ ​and​ ​General​ ​Questions

    New Compound Might Limit Muscle Damage in MD Type 1

    Posted by md-forums-moderator on April 17, 2019 at 9:03 am

    Cugamycin, a small molecule compound targeting the disease-causing RNA repeats in people with myotonic dystrophy type 1 (DM1), showed promise in improving muscle defects — without evident side effects — in an early study using mouse and cell models, researchers report.

    Their study, “Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model,” was published in PNAS. Read a summary of this research here.

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