Treatment of Glycogen Storage Disease Type IV in Dilated Cardiomyopathy

When Glycogen storage disease type IV (GSD IV) presents with dilated cardiomyopathy and no hypertrophic cardiomyopathy, cardiac involvement requires a specific, evidence-based pharmacological approach distinct from other GSD IV phenotypes.

Clinical scenario: GSD IV with confirmed dilated cardiomyopathy (DCM) and no hypertrophic cardiomyopathy. This phenotype calls for targeted cardiac pharmacotherapy to address the dilated, non-hypertrophic pattern of myocardial involvement.

Treatment approach — partial overview

In this setting, cardiac pharmacotherapy acting on adrenergic or renin-angiotensin pathways is considered reasonable. The specific agent selection and the complete clinical decision algorithm are available in the full structured protocol.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1016/j.ymgme.2023.107525

For individuals with DCM, use of either β-blockers (either β1-selective, or combination non-selective β1/α1 blocker, such as carvedilol), or either angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs), is reasonable.

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