EP 126: Patient-led research for ultra-rare disease drug development with Julia Taravella, Executive Director of Rare Trait Hope Fund
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0:00 Introduction0:25 Julia’s personal experience with ultra-rare diseases within her family10:25 The importance of increasing accessibility to genetic testing across populations to learn more about ultra-rare diseases14:00 The origin story of Rare Trait Hope Fund23:50 Current research and next steps in developing potential gene therapies for aspartylglucosaminuria32:20 How best to applykey insights and best practices to improve the approach to ultra-rare disease treatment development38:30 Next steps for Rare Trait Hope Fund and how people can help with Julia’s mission44:00 Julia’s advice for families who are advocating for rare disease research and therapy development 46:50 Closing remarksFor more information on Rare Trait Hope Fund, please visit: https://www.raretrait.com/).