
Recorded live at UConn’s 2026 Rare Disease Symposium, we reveal the grueling work of the rare journey, from battling medical dismissal and insurance denials to the fierce advocacy required.
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#392 Your DNA Is Not Static: Roxanne Khamsi on Mutation & Mosaicism

#391 Living with FSGS: A Genetic Kidney Disease Journey

#390 Prince, Mayte Garcia, and Their Son Amiir’s Pfeiffer Syndrome Type 2 Story

#389 From Natural History to Gene Therapy: The Future of Danon Disease Research
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