International Niemann–Pick Disease Alliance
“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...
Want to join the NNPDF Family Support & Medical Conference but can’t be there in person? VIRTUAL Conference registration is available! Cost is per family (includes multiple devices) and includes main sessions. Check out the agenda at t.ly/agenda2022 for all LiveStream...
Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD Paris, June 28, 2022. The European Commission (EC) has approved Xenpozyme® (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of...