EP 259: A patient community driving awareness and research for Danon disease with Jenny Hsieh of the Danon Foundation

SummaryThis week on The Genetics Podcast, Patrick is joined by Jenny Hsieh, Co-Founder and President of the Danon Foundation. They discuss how Danon disease affects males and females, why it is so often misdiagnosed, the current therapy landscape and research priorities, and how families can make informed decisions about gene therapy trials.Show Notes0:00 Intro to The Genetics Podcast00:59 Welcome to Jenny 01:28 An overview of Danon disease02:34 Sex differences in Danon disease and severe disease in females05:15 Why Danon disease is misdiagnosed and how it may be spotted earlier08:50 Therapy pipeline for Danon and the Foundation's global patient coordination13:46 Origin story of the Danon Foundation18:10 Research priorities and the burden of Danon disease beyond the heart22:39 Raising genetic testing awareness among ophthalmologists and cardiologists for Danon disease25:20 Recognizing broader Danon symptoms and launching the first Danon Awareness Day29:37 Informed consent and trial literacy for families weighing gene therapy33:14 Patient-friendly trial experiences36:19 Ways to support Danon research37:37 Closing remarksFind out moreDanon Foundation