PYGM

Chr 11

glycogen phosphorylase, muscle associated

Also known as: GSD5

This gene encodes a muscle enzyme involved in glycogenolysis. Highly similar enzymes encoded by different genes are found in liver and brain. Mutations in this gene are associated with McArdle disease (myophosphorylase deficiency), a glycogen storage disease of muscle. Alternative splicing results in multiple transcript variants.[provided by RefSeq, Sep 2009]

GeneReviewsResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

UniProtGlycogen storage disease 5

Clinical highlights

Gene-disease validity (ClinGen)
glycogen storage disease V · ARDefinitivesufficient evidence for diagnostic panels
0
Active trials
50
Pubs (1 yr)
P/LP submissions
P/LP missense
0.87
LOEUF
Multiple*
Mechanism· predicted
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GeneReview available — PYGM
Authoritative clinical overview · Recommended first read
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Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint?
0.87LOEUF
pLI 0.000
Z-score 2.22
OE 0.63 (0.470.87)
Tolerant

Typical tolerance to LoF variation

Missense Constraint?
0.05Z-score
OE missense 0.99 (0.921.07)
519 obs / 522.0 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios?
LoF OE?0.63 (0.470.87)
00.351.4
Missense OE?0.99 (0.921.07)
00.61.4
Synonymous OE?1.05
01.21.6
LoF obs/exp: 27 / 42.6Missense obs/exp: 519 / 522.0Syn Z: -0.52

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

PYGM · protein map & ClinVar variants

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

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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