International Niemann–Pick Disease Alliance
– Phase 3 trial met the primary endpoint and key secondary endpoints showing high statistical significance – IB1001 showed a clinically meaningful improvement in symptoms, functioning, quality of life, and cognition in both pediatric and adult patients with NPC...
“Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...
“Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...
Leiden, The Netherlands, January 5, 2023 – Azafaros B.V. today announced that it has received Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) to conduct a clinical Phase 2 trial for its lead...
IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288). Enrolment for IB1001-301 commenced...
– Data published in official journal of the Society for Inherited Metabolic Disorders, Molecular Genetics and Metabolism – Published trial data show that Trappsol® Cyclo™ overcomes the NPC1 defect by removing trapped cholesterol from cells both systemically and in the...