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A reprieve from hemophilia A, but for how long?

The agency’s ‘no-go’ for BioMarin’s hemophilia A gene therapy came as a surprise, but reflects outstanding questions as to the durability of the treatment.

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Sheridan, C. A reprieve from hemophilia A, but for how long?. Nat Biotechnol 38, 1107–1109 (2020). https://doi.org/10.1038/s41587-020-0693-y

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