News

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...

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

Professional delegates and Industry Representatives can register now for our NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Disease 2022!

Families Registration for the much-loved NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Disease 2022 is now officially open!

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