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 12. Congenital myasthenic syndrome
 [ 4 clinical trials,    5 drugs(DrugBank: 3 drugs),    3 target genes / 11 target pathways

Searched query = "Congenital myasthenic syndrome", "End plate acetylcholine receptor deficiency", "Sodium channel myasthenia", "End plate acetylcholine esterase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
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PhaseCountries
1NCT03062631February 20, 201711 June 2018Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton SyndromeTreatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton SyndromeCongenital Myasthenic Syndrome;Lambert-Eaton Myasthenic SyndromeDrug: 3,4-DiaminopyridineRicardo MaselliJacobus PharmaceuticalNot recruiting3 Months75 YearsAllPhase 1United States
2NCT02189720August 20142 September 2019Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS)An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)Congenital Myasthenic SyndromeDrug: Amifampridine PhosphateCatalyst Pharmaceuticals, Inc.Not recruiting2 YearsN/AAllPhase 2United States
3NCT01203592September 201018 January 2016Efficacy of Albuterol in the Treatment of Congenital Myasthenic SyndromesEfficacy of Albuterol in the Treatment of Congenital Myasthenic SyndromesCongenital Myasthenic SyndromeDrug: AlbuterolMayo ClinicNot recruiting2 YearsN/ABoth21Phase 1United States
4NCT00872950June 200125 June 20183,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)Open Label Trial Of 3,4-Diaminopyridine In Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)Lambert-Eaton Myasthenic Syndrome;Congenital Myasthenic SyndromeDrug: 3,4-DIAMINOPYRIDINE;Drug: 3,4-DiaminopyridineLahey ClinicRecruiting18 YearsN/AAll25N/AUnited States