12. Congenital myasthenic syndrome
5 clinical trials,   7 drugs   (DrugBank: 3 drugs),   5 drug target genes,   12 drug target pathways

Searched query = "Congenital myasthenic syndrome", "End-plate acetylcholine receptor deficiency", "Sodium channel myasthenia", "End-plate acetylcholine esterase deficiency", "Dok-7 myasthenia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02189720
(ClinicalTrials.gov)
July 20145/7/2014Expanded 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.NULLNo longer available2 YearsN/AAllUnited States