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Central Nervous System Involvement as Outcome Measure for Clinical Trials Efficacy in Myotonic Dystrophy Type 1

Increasing evidences indicate that in Myotonic Dystrophy type 1 (DM1 or Steinert disease), an autosomal dominant multisystem disorder caused by a (CTG)n expansion in DMPK gene on chromosome 19q13. 3, is the most common form of inherited muscular dystrophy in adult patients with a global prevalence o...

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Principais autores: Costanza Simoncini, Giulia Spadoni, Elisa Lai, Lorenza Santoni, Corrado Angelini, Giulia Ricci, Gabriele Siciliano
Format: Artigo
Sprog:Inglês
Udgivet: Frontiers Media S.A. 2020-10-01
Serier:Frontiers in Neurology
Fag:
myotonic dystrophy type 1
central nervous system
clinical trials
neuropsychological outcome measures
neuroimaging
Online adgang:https://www.frontiersin.org/article/10.3389/fneur.2020.00624/full
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