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Presynaptic congenital myasthenic syndrome with altered synaptic vesicle homeostasis linked to compound heterozygous sequence variants in RPH3A

BACKGROUND: Monogenic defects of synaptic vesicle (SV) homeostasis have been implicated in many neurologic diseases, including autism, epilepsy, and movement disorders. In addition, abnormal vesicle exocytosis has been associated with several endocrine dysfunctions. METHODS: We report an 11 year old...

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Detalhes bibliográficos
Publicado no:Mol Genet Genomic Med
Main Authors: Maselli, Ricardo A., Vázquez, Jessica, Schrumpf, Leah, Arredondo, Juan, Lara, Marian, Strober, Jonathan B., Pytel, Peter, Wollmann, Robert L., Ferns, Michael
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2018
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6014458/
https://ncbi.nlm.nih.gov/pubmed/29441694
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mgg3.370
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