12. 先天性筋無力症候群
［臨床試験数：5，薬物数：7（DrugBank：3），標的遺伝子数：5，標的パスウェイ数：12］
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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02189720 (ClinicalTrials.gov) | July 2014 | 5/7/2014 | Expanded 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 Syndrome | Drug: Amifampridine Phosphate | Catalyst Pharmaceuticals, Inc. | NULL | No longer available | 2 Years | N/A | All | United States |