Literature DB >> 27706425

Clinical and genetic basis of congenital myasthenic syndromes.

Paulo Victor Sgobbi de Souza1, Gabriel Novaes de Rezende Batistella1, Valéria Cavalcante Lino1, Wladimir Bocca Vieira de Rezende Pinto1, Marcelo Annes1, Acary Souza Bulle Oliveira1.   

Abstract

Neuromuscular junction disorders represent a wide group of neurological diseases characterized by weakness, fatigability and variable degrees of appendicular, ocular and bulbar musculature involvement. Its main group of disorders includes autoimmune conditions, such as autoimmune acquired myasthenia gravis and Lambert-Eaton syndrome. However, an important group of diseases include congenital myasthenic syndromes with a genetic and sometimes hereditary basis that resemble and mimick many of the classic myasthenia neurological manifestations, but also have different presentations, which makes them a complex clinical, therapeutic and diagnostic challenge for most clinicians. We conducted a wide review of congenital myasthenic syndromes in their clinical, genetic and therapeutic aspects.

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Year:  2016        PMID: 27706425     DOI: 10.1590/0004-282X20160106

Source DB:  PubMed          Journal:  Arq Neuropsiquiatr        ISSN: 0004-282X            Impact factor:   1.420


  8 in total

1.  Collagen XIII Is Required for Neuromuscular Synapse Regeneration and Functional Recovery after Peripheral Nerve Injury.

Authors:  Zarin Zainul; Anne Heikkinen; Hennariikka Koivisto; Iina Rautalahti; Mika Kallio; Shuo Lin; Heli Härönen; Oula Norman; Markus A Rüegg; Heikki Tanila; Taina Pihlajaniemi
Journal:  J Neurosci       Date:  2018-04-06       Impact factor: 6.167

2.  Novel compound heterozygous variants in the GFPT1 gene leading to rare limb-girdle congenital myasthenic syndrome with rimmed vacuoles.

Authors:  Yanyan Ma; Ting Xiong; Guohua Lei; Jiaqi Ding; Rui Yang; Zunbo Li; Jun Guo; Dingguo Shen
Journal:  Neurol Sci       Date:  2021-01-13       Impact factor: 3.307

Review 3.  How to Spot Congenital Myasthenic Syndromes Resembling the Lambert-Eaton Myasthenic Syndrome? A Brief Review of Clinical, Electrophysiological, and Genetics Features.

Authors:  Paulo José Lorenzoni; Rosana Herminia Scola; Claudia Suemi Kamoi Kay; Lineu Cesar Werneck; Rita Horvath; Hanns Lochmüller
Journal:  Neuromolecular Med       Date:  2018-04-25       Impact factor: 3.843

4.  Congenital myasthenic syndrome due to rapsyn deficiency: A case report with a new mutation and compound heterozygosity.

Authors:  Ivan O Espinoza; Carolina Reynoso; Giulliana Chávez; Andrew G Engel
Journal:  Medwave       Date:  2019-06-04

5.  Congenital myasthenic syndromes.

Authors:  Josef Finsterer
Journal:  Orphanet J Rare Dis       Date:  2019-02-26       Impact factor: 4.123

6.  The First Case of Congenital Myasthenic Syndrome Caused by a Large Homozygous Deletion in the C-Terminal Region of COLQ (Collagen Like Tail Subunit of Asymmetric Acetylcholinesterase) Protein.

Authors:  Nicola Laforgia; Lucrezia De Cosmo; Orazio Palumbo; Carlotta Ranieri; Michela Sesta; Donatella Capodiferro; Antonino Pantaleo; Pierluigi Iapicca; Patrizia Lastella; Manuela Capozza; Federico Schettini; Nenad Bukvic; Rosanna Bagnulo; Nicoletta Resta
Journal:  Genes (Basel)       Date:  2020-12-18       Impact factor: 4.096

Review 7.  Novel copy number variation of COLQ gene in a Moroccan patient with congenital myasthenic syndrome: a case report and review of the literature.

Authors:  Youssef El Kadiri; Ilham Ratbi; Abdelaziz Sefiani; Jaber Lyahyai
Journal:  BMC Neurol       Date:  2022-08-05       Impact factor: 2.903

8.  Congenital myasthenic syndrome caused by a frameshift insertion mutation in GFPT1.

Authors:  Szabolcs Szelinger; Jonida Krate; Keri Ramsey; Samuel P Strom; Perry B Shieh; Hane Lee; Newell Belnap; Chris Balak; Ashley L Siniard; Megan Russell; Ryan Richholt; Matt De Both; Ana M Claasen; Isabelle Schrauwen; Stanley F Nelson; Matthew J Huentelman; David W Craig; Samuel P Yang; Steven A Moore; Kumaraswamy Sivakumar; Vinodh Narayanan; Sampathkumar Rangasamy
Journal:  Neurol Genet       Date:  2020-06-30
  8 in total

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