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 12. 先天性筋無力症候群 [臨床試験数:4,薬物数:5(DrugBank:3),標的遺伝子数:1,標的パスウェイ数:3] 

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. 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"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
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