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Tell your story. Tell your story again. Then tell it again.   That’s the beginning of advocacy for rare disease.  “You have to be assertive and speak up. You don’t have time to waste!” advocate [...]

Kristen Steele knows a thing or two about telling her story and getting what she needs to be her best. The 22-year-old from Council Bluffs, Iowa, is a licensed massage therapist in Iowa and [...]

Known as a pioneer in gene therapy, Jean Bennett, MD, PhD, surveyed her audience of patients and families living with Leber congenital amaurosis and declared: “YOU are all the pioneers!” Dr. [...]