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How Love Combined with Science Leads to Hope for Rare Disease
Episode 89

How Love Combined with Science Leads to Hope for Rare Disease

There are over 7000 rare diseases identified in the U.S. alone. Many of these diseases affect children, leaving families desperate for answers. In this episode, we talk with one of those families and the biotech company offering them new hope. We also talk with an expert on policy that has jump-started R&D for rare diseases.

I AM BIO · Courtney Dion President and Co-founder The Dion Foundation for Children with Rare Disease, Joe Dion Co-founder and Treasurer The Dion Foundation for Children with Rare Disease, Stéphane Degove Co-founder & CEO Atamyo Therapeutics, Jamie Sullivan VP Policy EveryLife Foundation for Rare Diseases

October 22, 202427m 5s

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Show Notes

There are over 7000 rare diseases identified in the U.S. alone. Many of these diseases affect children, leaving families desperate for answers. In this episode, we talk with one of those families and the biotech company offering them new hope. We also talk with an expert on policy that has jump-started R&D for rare diseases.

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Topics

patient advocacyeverylife foundationmuscujlar dystrophylgmd 2clgmddion foundationstéphane degovecourtney dionrare pediatric disease designation and priority review voucher programlimb girdle muscular dystrophy type 2cgene mutationeverylifejamie sullivanprvpatient advocateatamyo therapeuticsadeno-associated vires vectorrare diseasesata 200dmddion foundation for children with rare diseaserare diseasejoe dionaavneuromuscular disorderfdagene therapy