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Researchers welcome $3.5-million haemophilia gene therapy — but questions remain

The world’s most expensive drug has the potential to save lives. But it cannot treat the most common form of the disease.
  1. Miryam Naddaf
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On 22 November, the US Food and Drug Administration (FDA) approved the first gene therapy for the genetic blood-clotting disorder haemophilia B — a one-time treatment that costs US$3.5 million.

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Nature 612, 388-389 (2022)

doi: https://doi.org/10.1038/d41586-022-04327-7

References

  1. Nathwani, A. C. et al. Blood 132 (S1), 491 (2018).

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