Condition (MeSH)
Myasthenic Syndromes, Congenital
ClinicalTrials.gov lists 19 trials for Myasthenic Syndromes, Congenital or a narrower condition beneath it. argenx has the most among industry and government lead sponsors, with 3.
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Trials per phase
19 of the 19 trials are tagged with Myasthenic Syndromes, Congenital itself; the rest are tagged with a narrower condition beneath it.
When did Myasthenic Syndromes, Congenital trials start? (9)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 3
- Observational
Top of scale: 3
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2025 | 2 |
| 2024 | 3 |
| 2022 | 1 |
| 2021 | 1 |
| 2016 | 1 |
| 2010 | 2 |
| 2009 | 1 |
| 2007 | 1 |
No forecast is made for this condition: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Myasthenic Syndromes, Congenital trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 5 |
| Phase 1/2 | 1 |
| Phase 3 | 2 |
| Observational | 6 |
| Expanded access | 5 |
How many Myasthenic Syndromes, Congenital trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 4 |
| Active, not recruiting | 1 |
| Completed | 6 |
| Approved for marketing | 2 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Myasthenic Syndromes, Congenital trials? (4)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| argenx | 3 |
| Catalyst Pharmaceuticals, Inc. | 2 |
| Amplo Biotechnology | 1 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 1 |
Which drugs are being tested for Myasthenic Syndromes, Congenital? (4)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| 3, 4-Diaminopyridine | 6 |
| ARGX-119 | 2 |
| Ephedrine | 1 |
| Albuterol | 1 |
Interventions as named in the registry (3)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| AlloEx exosomes | 1 |
| Exosome Secretome | 1 |
| AMP-101 | 1 |
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Myasthenic Syndromes, Congenital trials report? (4)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Hypotension | 1 | 1 |
| Parainfluenzae virus infection | 1 | 1 |
| Pyrexia | 1 | 1 |
| Rhinovirus infection | 1 | 1 |
Which publications cover Myasthenic Syndromes, Congenital? (10)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Developing outcome measures of disease activity in pediatric myasthenia. | Muscle & nerve | 2021 | Yes |
| Congenital myasthenic syndromes: where do we go from here? | Neuromuscular disorders : NMD | 2021 | Yes |
| Prevalence and genetic subtypes of congenital myasthenic syndromes in the pediatric population of Slovenia. | European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society | 2020 | Yes |
| Italian recommendations for diagnosis and management of congenital myasthenic syndromes. | Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology | 2019 | Yes |
| Salbutamol modifies the neuromuscular junction in a mouse model of ColQ myasthenic syndrome. | Human molecular genetics | 2019 | Yes |
Showing 5 of 10 publications.
Prevalence (6)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Congenital myasthenic syndrome | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
| Congenital myasthenic syndrome | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Congenital myasthenic syndrome | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Congenital myasthenic syndrome | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Congenital myasthenic syndrome | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Congenital myasthenic syndrome | 590 | Exact | Orphadata Science knowledge files, Product 1 |
| Congenital myasthenic syndrome | 590 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.