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    Open Access

    Anakinra in Sanfilippo syndrome: a phase 1/2 trial

    Sanfilippo syndrome is a fatal childhood neurodegenerative disorder involving neuroinflammation among multiple pathologies. We hypothesized that anakinra, a recombinant interleukin-1 receptor antagonist, could...

    Lynda E. Polgreen, Agnes H. Chen, Youngju Pak, Anna Luzzi… in Nature Medicine (2024)

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