glycogen storage disease IXb

MeSH: C563008ORPHA: 79240

Overview

Glycogen storage disease (GSD) due to liver and muscle phosphorylase kinase (PhK) deficiency is a benign inborn error of glycogen metabolism. It is the mildest form of GSD due to PhK deficiency (see this term).

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with glycogen storage disease IXb, sourced from HPO and Orphanet clinical annotations.

HypertriglyceridemiaHepatomegalyPostnatal growth retardationAbnormal circulating enzyme concentration or activityHepatic fibrosisElevated circulating hepatic transaminase concentrationHypercholesterolemiaFasting hypoglycemiaPolycystic ovariesDelayed speech and language developmentIrregular menstruationOligomenorrheaHypotoniaCirrhosisSplenomegalyRenal tubular acidosisRecurrent hypoglycemiaDelayed gross motor developmentRecurrent infectionsSkeletal muscle atrophyElevated circulating creatine kinase concentrationProgressive muscle weaknessLimb-girdle muscle weaknessMyalgiaMuscle spasmExercise intolerancePelvic girdle muscle weaknessShort statureMild global developmental delayFatigueKetotic hypoglycemiaDysmenorrheaOsteoporosisHepatocellular carcinomaAnemiaVomitingDiarrheaNauseaMyoglobinuriaLactic acidosisRhabdomyolysisIncreased body weightHepatocellular adenoma

Classification & Codes

MeSH Code

C563008

Orphanet Code

ORPHA:79240
glycogen storage disease IXb
MeSHC563008
OrphanetORPHA:79240
Treatments0 drug(s)
Symptoms on record43 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO