EPM2A

Chr 6AR

EPM2A glucan phosphatase, laforin

Also known as: EPM2, MELF, MELF2

The protein is a dual-specificity phosphatase that prevents glycogen hyperphosphorylation by acting on complex carbohydrates, thereby avoiding formation of insoluble glycogen aggregates. Mutations cause Lafora disease (myoclonic epilepsy of Lafora type 1), a rare neurodegenerative disorder with adult onset characterized by myoclonus epilepsy and accumulation of insoluble glycogen-derived particles called Lafora bodies. Inheritance is autosomal recessive.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM
MultiplemechanismARLOEUF 1.341 OMIM phenotype
Clinical SummaryEPM2A
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Gene-Disease Validity (ClinGen)
Lafora disease · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
48 unique Pathogenic / Likely Pathogenic· 190 VUS of 400 total submissions
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GeneReview available — EPM2A
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.34LOEUF
pLI 0.000
Z-score 0.73
OE 0.77 (0.461.34)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.26Z-score
OE missense 1.06 (0.931.20)
165 obs / 155.9 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.77 (0.461.34)
00.351.4
Missense OE1.06 (0.931.20)
00.61.4
Synonymous OE1.27
01.21.6
LoF obs/exp: 9 / 11.7Missense obs/exp: 165 / 155.9Syn Z: -1.64
DN
0.6066th %ile
GOF
0.6931th %ile
LOF
0.53top 25%

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and loss-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to gain-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median
LOF63% of P/LP variants are LoF

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

400 submitted variants in ClinVar

Classification Summary

Pathogenic30
Likely Pathogenic18
VUS190
Likely Benign138
Benign14
Conflicting7
30
Pathogenic
18
Likely Pathogenic
190
VUS
138
Likely Benign
14
Benign
7
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
21
1
8
0
30
Likely Pathogenic
9
8
1
0
18
VUS
4
178
8
0
190
Likely Benign
2
14
36
86
138
Benign
0
0
14
0
14
Conflicting
7
Total362016786397

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

EPM2A · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Lafora disease.
Turnbull J et al.·Epileptic Disord
2016Review
Polyglucosan storage myopathies.
Hedberg-Oldfors C et al.·Mol Aspects Med
2015Review
Lafora disease: from genotype to phenotype.
Parihar R et al.·J Genet
2018Review
FDG-PET assessment and metabolic patterns in Lafora disease.
Muccioli L et al.·Eur J Nucl Med Mol Imaging
2020
Top 5 results · since 2015Search PubMed ↗