Thank you for donating!

You can donate using the following services.


  1. 03.10.22

    301 NPC UK Recruitment Update - IntraBio

    IntraBio Ltd. is pleased to share that the Phase III pivotal trial with N-acetyl-L-leucine (IB1001-301) clinical trial has enrolled over 30% of the target number of patients in the first month of recruitment. The study, which investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), will enroll a total of approximately 52 patients across all international sites. Since recruitment commenced in September 2022, sixteen (20) patients have been screened...

    Read more
  2. 08.09.22

    Drug Repurposing for Rare Diseases 2022 is shifting its programme to match the new drug repurposing landscape.

    Read more
  3. 28.06.22

    Press Release: Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD

    Read more
  4. 15.06.22

    IntraBio: IB1001-301 (UK Recruitment)

    Read more
  5. 30.05.22

    Mandos NPC1 Community Newsletter, May 2022

    Read more