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My daughter has a rare disease. We shouldn’t have had to leave the US to save her life.
My daughter has a rare disease. We shouldn’t have had to leave the US to save her life.
My daughters were diagnosed with a rare genetic disorder. We had to leave the country to give one a chance at treatment.
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← FDA approves new gene therapy treatment for kids priced at $4.25 million
A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access →
