Key result
CACNA1S variant p.Leu1243Val links to hypokalemic periodic paralysis in a single patient.
Why the study?
Most pathogenic CACNA1S mutations in hypokalemic periodic paralysis affect arginine residues in S4 voltage-sensor domains, but other variants remain poorly understood.
This case expands the mutational spectrum of hypokalemic periodic paralysis by highlighting a possible genotype-phenotype link involving the rare CACNA1S p.Leu1243Val variant.
Warrants no change in hypokalemic periodic paralysis care; extends CACNA1S spectrum but remains hypothesis-generating.
Background Hypokalemic periodic paralysis (HypoPP) is a rare skeletal muscle channelopathy, most often caused by mutations in CACNA1S or SCN4A . Most pathogenic CACNA1S mutations affect arginine residues in S4 voltage‐sensor domains, but other variants remain poorly understood. Case Presentation I describe a 30‐year‐old Caucasian woman with recurrent paralytic episodes and hypokalemia (2.1–2.3 mmol/L), triggered by stress and carbohydrate‐rich meals. Genetic testing revealed heterozygosity for CACNA1S c.3727C > G (p.Leu1243Val), a variant of uncertain significance not previously associated with pathogenicity. Her phenotype was consistent with HypoPP. Treatment with spironolactone and acetazolamide reduced episode frequency, although the latter caused intolerable side effects, particularly tachypnea; she was later approved for dichlorphenamide. During one hospitalization, she also developed transient hypophosphatemia and hypokalemia, consistent with her HypoPP picture. Kidney function and imaging were normal. Family history revealed electrolyte disturbances in her grandfather. Conclusions This case highlights a possible genotype–phenotype link involving CACNA1S p.Leu1243Val. Continued reporting of such cases is essential for variant reclassification and for improving recognition of metabolic shifts during HypoPP attacks.
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Mark Abi Nader (2026) studied this question. The CACNA1S variant p.Leu1243Val was identified in a patient with hypokalemic periodic paralysis, highlighting a potential genotype-phenotype link.
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