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  1. 13.03.24

    Azafaros: Completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

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  2. 06.03.24

    NPUK Statement: NICE Olipudase Alfa Decision

    Yesterday, we were greatly disappointed to learn that the UK's National Institute for Health and Care Excellence (NICE) has decided not to recommend Sanofi's Xenpozyme (olipudase alfa) as a treatment for acid sphingomyelinase deficiency (ASMD; Niemann-Pick Disease) in people with type AB or type B...

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  3. 08.02.24

    2024 Regional Family Days: Save the Dates!

    We're pleased to announce this year's Regional Family Days, designed to bring our community together for support and solidarity. Here's the schedule...

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  4. 07.02.24

    IntraBio: NEJM Publishes Expert Perspective Editoral on IB1001

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  5. 02.02.24

    NEJM publishes IB1001-301 Trial Results

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