Literature DB >> 29696584

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

Paulo José Lorenzoni1,2, Rosana Herminia Scola3, Claudia Suemi Kamoi Kay3, Lineu Cesar Werneck3, Rita Horvath4, Hanns Lochmüller5,6.   

Abstract

Congenital myasthenic syndromes (CMS) are heterogeneous genetic diseases in which neuromuscular transmission is compromised. CMS resembling the Lambert-Eaton myasthenic syndrome (CMS-LEMS) are emerging as a rare group of distinct presynaptic CMS that share the same electrophysiological features. They have low compound muscular action potential amplitude that increment after brief exercise (facilitation) or high-frequency repetitive nerve stimulation. Although clinical signs similar to LEMS can be present, the main hallmark is the electrophysiological findings, which are identical to autoimmune LEMS. CMS-LEMS occurs due to deficits in acetylcholine vesicle release caused by dysfunction of different components in its pathway. To date, the genes that have been associated with CMS-LEMS are AGRN, SYT2, MUNC13-1, VAMP1, and LAMA5. Clinicians should keep in mind these newest subtypes of CMS-LEMS to achieve the correct diagnosis and therapy. We believe that CMS-LEMS must be included as an important diagnostic clue to genetic investigation in the diagnostic algorithms to CMS. We briefly review the main features of CMS-LEMS.

Entities:  

Keywords:  Congenital myasthenic syndromes; Lambert–Eaton myasthenic syndrome

Mesh:

Substances:

Year:  2018        PMID: 29696584     DOI: 10.1007/s12017-018-8490-1

Source DB:  PubMed          Journal:  Neuromolecular Med        ISSN: 1535-1084            Impact factor:   3.843


  33 in total

Review 1.  Membrane fusion: SNAREs and regulation.

Authors:  J Malsam; S Kreye; T H Söllner
Journal:  Cell Mol Life Sci       Date:  2008-09       Impact factor: 9.261

2.  Evidence that agrin directly influences presynaptic differentiation at neuromuscular junctions in vitro.

Authors:  J A Campagna; M A Ruegg; J L Bixby
Journal:  Eur J Neurosci       Date:  1997-11       Impact factor: 3.386

Review 3.  The Synaptic Vesicle Release Machinery.

Authors:  Josep Rizo; Junjie Xu
Journal:  Annu Rev Biophys       Date:  2015       Impact factor: 12.981

Review 4.  The Increasing Genetic and Phenotypical Diversity of Congenital Myasthenic Syndromes.

Authors:  Grace McMacken; Angela Abicht; Teresinha Evangelista; Sally Spendiff; Hanns Lochmüller
Journal:  Neuropediatrics       Date:  2017-05-15       Impact factor: 1.947

Review 5.  Synaptic basal lamina-associated congenital myasthenic syndromes.

Authors:  Ricardo A Maselli; Juan Arredondo; Michael J Ferns; Robert L Wollmann
Journal:  Ann N Y Acad Sci       Date:  2012-12       Impact factor: 5.691

6.  Non-paraneoplastic Lambert-Eaton myasthenic syndrome: a brief review of 10 cases.

Authors:  Paulo J Lorenzoni; Rosana H Scola; Cláudia S Kamoi Kay; Sérgio F Parolin; Lineu C Werneck
Journal:  Arq Neuropsiquiatr       Date:  2010-12       Impact factor: 1.420

Review 7.  Congenital myasthenic syndromes: progress over the past decade.

Authors:  Andrew G Engel; Kinji Ohno; Steven M Sine
Journal:  Muscle Nerve       Date:  2003-01       Impact factor: 3.217

8.  Agrin mutations lead to a congenital myasthenic syndrome with distal muscle weakness and atrophy.

Authors:  Sophie Nicole; Amina Chaouch; Torberg Torbergsen; Stéphanie Bauché; Elodie de Bruyckere; Marie-Joséphine Fontenille; Morten A Horn; Marijke van Ghelue; Sissel Løseth; Yasmin Issop; Daniel Cox; Juliane S Müller; Teresinha Evangelista; Erik Stålberg; Christine Ioos; Annie Barois; Guy Brochier; Damien Sternberg; Emmanuel Fournier; Daniel Hantaï; Angela Abicht; Marina Dusl; Steven H Laval; Helen Griffin; Bruno Eymard; Hanns Lochmüller
Journal:  Brain       Date:  2014-06-20       Impact factor: 13.501

9.  Loss of MUNC13-1 function causes microcephaly, cortical hyperexcitability, and fatal myasthenia.

Authors:  Andrew G Engel; Duygu Selcen; Xin-Ming Shen; Margherita Milone; C Michel Harper
Journal:  Neurol Genet       Date:  2016-09-08

Review 10.  The Multifaceted Role of SNARE Proteins in Membrane Fusion.

Authors:  Jing Han; Kristyna Pluhackova; Rainer A Böckmann
Journal:  Front Physiol       Date:  2017-01-20       Impact factor: 4.566

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