A Mother’s Mission to Develop Rare Disease Therapeutics

Julia Taravella shares the remarkable story of becoming a rare disease advocate and therapeutic developer after both of her children were diagnosed with aspartylglucosaminuria (AGU).

Through the Rare Trait Hope Fund, Julia has worked to advance the development of a gene therapy for AGU. In this conversation, she shares the realities of turning a family’s diagnosis into action, the progress being made toward treatment, and lessons that can inspire other rare disease communities working toward better therapies.

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Cure PDE Family Retreats

Our Family Retreats bring families from across the United States and Canada together for connection, education, research updates, and time with others who understand the PDE journey.

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Cure PDE brings families, researchers, clinicians, and advocates together throughout the year through in-person gatherings and educational conversations.

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