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COLQ-mutant Congenital Myasthenic Syndrome with Microcephaly: A Unique Case with Literature Review

Congenital Myasthenic Syndrome (CMS) is a group of inherited neuromuscular junction disorders caused by defects in several genes. Clinical features include delayed motor milestones, recurrent respiratory illnesses and variable fatigable weakness. The central nervous system involvement is typically n...

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Detalles Bibliográficos
Autores principales: Al-Muhaizea, Mohammad A., Al-Mobarak, Sulaiman Bazee
Formato: Online Artículo Texto
Lenguaje:English
Publicado: De Gruyter Open 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5518714/
https://www.ncbi.nlm.nih.gov/pubmed/28744372
http://dx.doi.org/10.1515/tnsci-2017-0011
Descripción
Sumario:Congenital Myasthenic Syndrome (CMS) is a group of inherited neuromuscular junction disorders caused by defects in several genes. Clinical features include delayed motor milestones, recurrent respiratory illnesses and variable fatigable weakness. The central nervous system involvement is typically not part of the CMS. We report here a Saudi girl with genetically proven Collagen Like Tail Subunit Of Asymmetric Acetylcholinesterase (COLQ) mutation type CMS who has global developmental delay, microcephaly and respiratory failure. We have reviewed the literature regarding COLQ-type CMS and to the best of our knowledge this is the first ever reported association of congenital myasthenia syndrome with microcephaly.