Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS.
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ABSTRACT:
SUBMITTER: Gourdon G
PROVIDER: S-EPMC5397409 | biostudies-literature | 2017
REPOSITORIES: biostudies-literature
Publications
Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS.
Gourdon Genevieve G Meola Giovanni G
Frontiers in cellular neuroscience 20170420
Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the past two decades, much progress has been made in understanding the mechanisms underlying the DM symptoms allowing development of new molecular therapeutic tools with the ultimate aim of curing the dis ...[more]
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