Thank you for donating!

You can donate using the following services.

News

  1. 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...

    Read more
  2. 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...

    Read more
  3. 07.02.24

    IntraBio: NEJM Publishes Expert Perspective Editoral on IB1001

    Read more
  4. 02.02.24

    NEJM publishes IB1001-301 Trial Results

    Read more
  5. 21.12.23

    Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

    Read more