International Niemann–Pick Disease Alliance
Combined company will operate as Cyclo Therapeutics with primary focus on advancing Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1) At closing combined Company will have approximately $13.7 million cash on hand to fund operations into 2H...
Celebrating Strength and Support during NPD Awareness Month… …let’s shine a bright light on the unsung heroes of our Niemann-Pick Disease (NPD) community this October, during Niemann-Pick Disease Awareness Month. We’re thrilled to introduce our “Sibling Spotlight” campaign, dedicated...
– Data published in official journal of Molecular Genetics and Metabolism Reports: Evidence of a change in cholesterol homeostasis in peripheral organs and the brain after the initial infusion Trappsol® Cyclo™ was detected in the CSF Completers had an 88.9% improvement...
– 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...