One shot drug to end Sicilian curse comes at $1.8 million cost

5 November 2019 - A new targeted gene therapy shows the promise and pitfalls of medical advances. ...

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Bluebird Bio CEO defends $1.8 million price for gene therapy — ‘It’s really thinking about it differently’

14 June 2019 - He says the one-time treatment is a game changer for patients, giving them a “lifelong benefit” while ...

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Affordable orphan drugs: a role for not-for-profit organisations

23 January 2017 - The success of the regulation on orphan medicinal products in the European Union is evidenced by the ...

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