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Danon Disease: Highly Penetrant, Extreme Morbid X linked Multisystemic Disorder
AAV9.4 LAMP-2 Improves Cardiac Histology and Function
What is Natural History & Why is it Important?
What is the Natural History of Danon Disease?
Who is included in the Danon Registry?
What are we learning from the Danon Registry?
Current efforts underway to better understand the Natural History of Danon Disease
How can you help?
Danon disease case study
Echocardiogram
Rare disease is a compelling argument for the academic medical center
LC32 as a biomarker for autophagy
Mitochondrial dysfunction
Overexpressing LAMP2
MRI findings in Danon disease
Danon disease gene therapy clinical trial
What is the prognosis following a heart transplant for Danon disease?
Closing remarks