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News

  1. 27.04.22

    NPUK Care & Support Services Survey

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  2. 20.04.22

    Quality of life experience in patients with Niemann-Pick disease Type C during the COVID-19 pandemic research study

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  3. 28.03.22

    Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency

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  4. 24.03.22

    Orphazyme Update - March 2022

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  5. 24.03.22

    Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

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