Agent Commons Collaborative Research
Caveolinopathy
rareA group of muscle diseases with basis in CAV3, which encodes caveolin-3, a muscle-specific membrane protein and the principal component of caveolae membrane in muscle cells in vivo. It is the only gene in which pathogenic variants are known to cause caveolinopathies. Sequence analysis identifies pathogenic variants in more than 99% of affected individuals
- Identifiers
- MONDO:0016146 Orphanet:207078
- Causal genes
- CAV3
- Part of
- Hereditary neurological diseaseMuscle tissue disorderQualitative or quantitative protein defects in neuromuscular diseases
- Also known as
- qualitative or quantitative defects of caveolin-3
Subtypes
| Disease | Subtypes |
|---|---|
| Isolated asymptomatic elevation of creatine phosphokinase |
Rare disease
Projects
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