Congenital myasthenic syndrome

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Congenital myasthenic syndrome due to defects in endplate development and maintenance

ORPHA:716825Etiological subtype

Also called Defect in endplate development and maintenance

What it is

Orphanet has not published a description for this disease yet. The identifiers, classification and cross-references below are still current.

Key facts

Inheritance
Autosomal recessive
Classified as
Etiological subtype

Recorded for the broader condition

Age of onset
Infancy, NeonatalPostsynaptic congenital myasthenic syndrome

Orphanet records these for the broader condition rather than for this specific form. They are a starting point, not a figure for this subtype — subtypes often differ, and some are defined by being rarer than the condition they sit under.

Genes

AGRNDisease-causing germline mutation(s)
DOK7Disease-causing germline mutation(s)
LRP4Disease-causing germline mutation(s)
MUSKDisease-causing germline mutation(s)

Cross-references

OMIM 254300OMIM 615120OMIM 616304OMIM 616325

Orphadata Science: Free access data from Orphanet. © INSERM 1999. Data version 2026-06-23. Available under CC BY 4.0. This is a reference, not medical advice.

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