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Ensuring a future for gene therapy for rare diseases - Nature Medicine

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Ensuring a future for gene therapy for rare diseases - Nature Medicine

Hematopoietic stem-cell gene therapy has proven to be an effective treatment for several primary immunodeficiencies, and yet companies in this space are withdrawing from the EU market. Technological and regulatory innovations and a change to cost–benefit models are needed so that rare disease patients can receive these life-saving medicines.

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Ensuring a future for gene therapy for rare diseases - Nature Medicine

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Ensuring a future for gene therapy for rare diseases - Nature Medicine

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