Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease
Systemic AAV9.LAMP2B injection reverses multiorgan dysfunction in a murine model of Danon disease
Clinical Profile of Cardiac Involvement in Danon Disease: A Multicenter European Registry
Danon disease: Review of natural history and recent advances
Danon disease: a phenotypic expression of LAMP-2 deficiency
Danon disease for the cardiologist: case report and review
Danon disease: a case report and literature review
Danon Disease: Entire LAMP2 Gene Deletion with Unusual Clinical Presentation
A novel LAMP2 mutation associated with severe cardiac hypertrophy and microvascular remodeling
Human iPSC model of Danon disease reveals LAMP2 haploinsufficiency and rescue by gene therapy