International Niemann-Pick Disease Alliance (INPDA)

Azafaros Receives FDA’s IND Clearance and Fast Track Designation for Lead Asset AZ-3102

Leiden, The Netherlands, January 5, 2023 – Azafaros B.V. today announced that it has received Investigational New Drug (IND)...
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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...
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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...
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Rare Disease Day (RDD) 2023
- Event date: 28.02.23
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