gapmer antisense

Antisense compound given to mice with a disorder resembling type 1 myotonic dystrophy safely corrected molecular defects and myotonia for a year

posted on August 2, 2012 - 11:01am
An experimental treatment for type 1 myotonic muscular dystrophy (MMD1, or DM1) has corrected several aspects of the disease in an MMD1 mouse model. A year after treatment with an experimental antisense oligonucleotide dubbed ASO 445236 ended, positive effects remained apparent in the mice.

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