These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
5. Advancing the Understanding of Vesicle-Associated Membrane Protein 1-Related Congenital Myasthenic Syndrome: Phenotypic Insights, Favorable Response to 3,4-Diaminopyridine, and Clinical Characterization of Five New Cases. Natera-de Benito D; Pugliese A; Polavarapu K; Guergueltcheva V; Tournev I; Todorova A; Afonso Ribeiro J; Fernández-Mayoralas DM; Ortez C; Martorell L; Estévez-Arias B; Matalonga L; Laurie S; Jou C; Lau J; Thompson R; Shen X; Engel AG; Nascimento A; Lochmüller H; Selcen D Pediatr Neurol; 2024 Aug; 157():5-13. PubMed ID: 38833907 [TBL] [Abstract][Full Text] [Related]
6. VAMP1-Related Congenital Myasthenic Syndrome: A Case Report and Literature Review. Yıldırım M; Yarenci GB; Genç MB; Uçar Çİ; Bayav S; Tekin MN; Bektaş Ö; Teber S Neuropediatrics; 2024 Jun; 55(3):200-204. PubMed ID: 38531369 [TBL] [Abstract][Full Text] [Related]
7. How to Spot Congenital Myasthenic Syndromes Resembling the Lambert-Eaton Myasthenic Syndrome? A Brief Review of Clinical, Electrophysiological, and Genetics Features. Lorenzoni PJ; Scola RH; Kay CSK; Werneck LC; Horvath R; Lochmüller H Neuromolecular Med; 2018 Jun; 20(2):205-214. PubMed ID: 29696584 [TBL] [Abstract][Full Text] [Related]
8. Congenital Myasthenic Syndrome Type 19 Is Caused by Mutations in COL13A1, Encoding the Atypical Non-fibrillar Collagen Type XIII α1 Chain. Logan CV; Cossins J; Rodríguez Cruz PM; Parry DA; Maxwell S; Martínez-Martínez P; Riepsaame J; Abdelhamed ZA; Lake AV; Moran M; Robb S; Chow G; Sewry C; Hopkins PM; Sheridan E; Jayawant S; Palace J; Johnson CA; Beeson D Am J Hum Genet; 2015 Dec; 97(6):878-85. PubMed ID: 26626625 [TBL] [Abstract][Full Text] [Related]
9. Biallelic loss of function variants in SYT2 cause a treatable congenital onset presynaptic myasthenic syndrome. Donkervoort S; Mohassel P; Laugwitz L; Zaki MS; Kamsteeg EJ; Maroofian R; Chao KR; Verschuuren-Bemelmans CC; Horber V; Fock AJM; McCarty RM; Jain MS; Biancavilla V; McMacken G; Nalls M; Voermans NC; Elbendary HM; Snyder M; Cai C; Lehky TJ; Stanley V; Iannaccone ST; Foley AR; Lochmüller H; Gleeson J; Houlden H; Haack TB; Horvath R; Bönnemann CG Am J Med Genet A; 2020 Oct; 182(10):2272-2283. PubMed ID: 32776697 [TBL] [Abstract][Full Text] [Related]
10. Identification of mutations in the MYO9A gene in patients with congenital myasthenic syndrome. O'Connor E; Töpf A; Müller JS; Cox D; Evangelista T; Colomer J; Abicht A; Senderek J; Hasselmann O; Yaramis A; Laval SH; Lochmüller H Brain; 2016 Aug; 139(Pt 8):2143-53. PubMed ID: 27259756 [TBL] [Abstract][Full Text] [Related]
11. The Neuromuscular Junction and Wide Heterogeneity of Congenital Myasthenic Syndromes. Rodríguez Cruz PM; Palace J; Beeson D Int J Mol Sci; 2018 Jun; 19(6):. PubMed ID: 29874875 [TBL] [Abstract][Full Text] [Related]
12. Congenital Myasthenic Syndromes in 2018. Engel AG Curr Neurol Neurosci Rep; 2018 Jun; 18(8):46. PubMed ID: 29892917 [TBL] [Abstract][Full Text] [Related]
13. Electrophysiological and morphological characterization of a case of autosomal recessive congenital myasthenic syndrome with acetylcholine receptor deficiency due to a N88K rapsyn homozygous mutation. Yasaki E; Prioleau C; Barbier J; Richard P; Andreux F; Leroy JP; Dartevelle P; Koenig J; Molgó J; Fardeau M; Eymard B; Hantaï D Neuromuscul Disord; 2004 Jan; 14(1):24-32. PubMed ID: 14659409 [TBL] [Abstract][Full Text] [Related]
14. Phenotypical and Myopathological Consequences of Compound Heterozygous Missense and Nonsense Variants in Della Marina A; Arlt A; Schara-Schmidt U; Depienne C; Gangfuß A; Kölbel H; Sickmann A; Freier E; Kohlschmidt N; Hentschel A; Weis J; Czech A; Grüneboom A; Roos A Cells; 2021 Dec; 10(12):. PubMed ID: 34943989 [TBL] [Abstract][Full Text] [Related]
15. Congenital myasthenic syndrome associated with epidermolysis bullosa caused by homozygous mutations in PLEC1 and CHRNE. Maselli RA; Arredondo J; Cagney O; Mozaffar T; Skinner S; Yousif S; Davis RR; Gregg JP; Sivak M; Konia TH; Thomas K; Wollmann RL Clin Genet; 2011 Nov; 80(5):444-51. PubMed ID: 21175599 [TBL] [Abstract][Full Text] [Related]
16. A common mutation (epsilon1267delG) in congenital myasthenic patients of Gypsy ethnic origin. Abicht A; Stucka R; Karcagi V; Herczegfalvi A; Horváth R; Mortier W; Schara U; Ramaekers V; Jost W; Brunner J; Janssen G; Seidel U; Schlotter B; Müller-Felber W; Pongratz D; Rüdel R; Lochmüller H Neurology; 1999 Oct; 53(7):1564-9. PubMed ID: 10534268 [TBL] [Abstract][Full Text] [Related]
17. Agrin mutations lead to a congenital myasthenic syndrome with distal muscle weakness and atrophy. Nicole S; Chaouch A; Torbergsen T; Bauché S; de Bruyckere E; Fontenille MJ; Horn MA; van Ghelue M; Løseth S; Issop Y; Cox D; Müller JS; Evangelista T; Stålberg E; Ioos C; Barois A; Brochier G; Sternberg D; Fournier E; Hantaï D; Abicht A; Dusl M; Laval SH; Griffin H; Eymard B; Lochmüller H Brain; 2014 Sep; 137(Pt 9):2429-43. PubMed ID: 24951643 [TBL] [Abstract][Full Text] [Related]
18. Animal Models of the Neuromuscular Junction, Vitally Informative for Understanding Function and the Molecular Mechanisms of Congenital Myasthenic Syndromes. Webster RG Int J Mol Sci; 2018 Apr; 19(5):. PubMed ID: 29710836 [TBL] [Abstract][Full Text] [Related]