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This study examines the natural course over time of Danon disease (a rare heart disease linked to the LAMP2 gene). It enrolled patients older than 6 years with a confirmed genetic mutation in the LAMP2 gene. It was a study without a phase and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria:
1. Patients older than 6 years old and any sex.
2. Patients with a confirmed genetic mutation in LAMP2. 3a. Living patients (who have not undergone heart transplantation or placement of a cardiac assist device):
* A minimum of 6 months of longitudinal data, including at least 2 echocardiograms or MRIs at least 6 months apart, prior to most recent follow-up visit or enrollment in Sponsor's gene therapy clinical trial.
3b. Deceased patients or living patients who underwent heart transplantation or cardiac assist device placement:
* At least 1 echocardiogram or MRI assessment prior to heart transplantation/ cardiac assist device placement or death
4\. Informed consent/assent by the patient and/or their legally authorized representative (if the patient is living at the time of enrollment).
Exclusion Criteria:
1\. Diagnosis of Danon disease without genetic testing confirmation.