Finding a CURE for rare genetic disorders

Get ready for one of our most meaningful conversations yet. Dr. Chloe sits down with Elizabeth DeLuca—the powerhouse advocate whose late-night PubMed searches, cross-country lab visits, and “help-the-babies” fund-raising campaigns helped push Capsida’s CAP-002 gene-therapy program all the way to FDA clearance. We unpack how this first-in-human trial aims to restore the missing STXBP1 protein, why the same technology could leapfrog into other rare epilepsies, and how a mother’s promise to her daughter is changing the face of science.Quick linksCapsida CAP-002 press release – details on the FDA-cleared trial and how it works: https://capsida.com/capsida-receives-fda-ind-clearance-for-its-first-in-class-iv-administered-gene-therapy-for-stxbp1-developmental-and-epileptic-encephalopathy/ capsida.comSTXBP1 Foundation – join the registry, learn about natural-history studies, or donate: https://www.stxbp1disorders.org/ STXBP1 FoundationSupport our mission by shopping the herbal lines that fund this podcast and Remy’s ongoing care: • Radical Roots Herbs – whole-plant, spagyric CBD formulas: https://RadicalRootsHerbs.com • Noxi Herbs – modular Chinese-herb system for women’s hormones: https://NoxiHerbs.comHeads-up: We speak candidly about seizures, neuro-degeneration, and child loss. Estimated annual mortality for STXBP1 remains ~3 %—every child is one too many—and that stark reality fuels Elizabeth’s drive and Dr. Chloe’s daily fight for her son, Remy.Listen, share, and stand with the rare-disease community that defines our world.See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.