A global network of non-profit organisations supporting those affected by Niemann-Pick diseases

IB1001-301 Recruitment Complete

IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on...

Cyclo Therapeutics Announces Publication of Phase 1 Data for Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1 (NPC1)

– Data published in official journal of the Society for Inherited Metabolic Disorders, Molecular Genetics and Metabolism – Published...

Xenpozyme (olipudase alfa) approved by FDA as first disease-specific treatment for ASMD (non-CNS manifestations)