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Sanofi wagers $400m on miRecule muscular dystrophy therapy

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The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

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