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Patient Input to Inform the Development of Central Nervous System Outcome Measures in Myotonic Dystrophy

Myotonic dystrophy type 1 (DM1) and myotonic dystrophy type 2 (DM2) are multisystem, genetic disorders caused by repeat expansions on chromosome 19 (DM1) and chromosome 3 (DM2). Although the effects of DM on the skeletal, cardiac, and smooth muscles, as well as the endocrine and central nervous syst...

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Detalhes bibliográficos
Publicado no:Ther Innov Regul Sci
Autor principal: White, Molly
Formato: Artigo
Idioma:Inglês
Publicado em: Springer International Publishing 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7458891/
https://ncbi.nlm.nih.gov/pubmed/31970692
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s43441-020-00117-3
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