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Danon disease in male patients: a prospective natural history study to augment understanding of the phenotype

Tarek Khedro1, Jennifer Attias2, Emily Eshraghian2

  • 1Department of Internal Medicine, Pennsylvania Hospital of the University of Pennsylvania Health System, Philadelphia, PA, USA.

Orphanet Journal of Rare Diseases
|October 22, 2025
PubMed
Abstract

No abstract available in PubMed .

Keywords:
Danon diseaseDifferential ability Scales-II (DAS-II)Lysosomal-associated membrane protein 2 (LAMP2)Nonischemic genetic cardiomyopathyNorth star ambulatory assessment (NSAA)Pediatric cardiac quality of life inventory (PCQLI)Pediatric quality of life inventory (PQLQ)Vineland adaptive behavior scales-3 (VABS-3)

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