Congenital Myasthenia (CM) (DBCOND0066674)

Identifiers

Synonyms

Congenital Myasthenic Syndrome / Myasthenic Syndromes, Congenital / Congenital Myasthenic Syndromes / Congenital Myasthenia / Congenital myasthenic syndrome (disorder)

Associated Data

Indicated Drugs and Targets

Drug	Description	Targets
Pyridostigmine	A cholinesterase inhibitor used for symptomatic treatment of myasthenia gravis and congenital myasthenic syndromes and to reverse neuromuscular blockade by nondepolarizing muscle relaxants.	Acetylcholinesterasetarget Cholinesterasetarget Acetylcholinesteraseenzyme Cholinesteraseenzyme Cytochrome P450 3A4enzyme Coagulation factor Xtarget

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT06436742	A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants with DOK7-Congenital Myasthenic Syndromes (CMS)	ARGX-119	treatment	1	recruiting
NCT01403402	Congenital Muscle Disease Study of Patient and Family Reported Medical Information	No drug interventions	Not Available	Not Available	recruiting
NCT05408702	Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes	No drug interventions	Not Available	Not Available	completed
NCT01474980	Pregnancy Outcomes in Congenital Myasthenie Syndrome	No drug interventions	Not Available	Not Available	completed
NCT06078553	A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)	No drug interventions	Not Available	Not Available	recruiting
NCT05687474	Baby Detect : Genomic Newborn Screening	No drug interventions	Not Available	Not Available	recruiting
NCT02189720	Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome	Amifampridine	Not Available	Not Available	no_longer_available
NCT03062631	Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia	Amifampridine	Not Available	Not Available	no_longer_available
NCT01203592	Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes	Albuterol	treatment	1	completed
NCT00872950	3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)	Amifampridine	Not Available	Not Available	approved_for_marketing
NCT06630650	A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes	No drug interventions	Not Available	Not Available	not_yet_recruiting
NCT02562066	Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes	Amifampridine	treatment	3	completed
NCT01765140	Treatment Use of 3,4-Diaminopyridine	Amifampridine	Not Available	Not Available	no_longer_available
NCT00541216	Ephedrine for the Treatment of Congenital Myasthenia	Ephedrine	treatment	1 / 2	unknown_status
NCT02012933	3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)	Amifampridine	Not Available	Not Available	no_longer_available