The Priority Review

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Azafaros

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  1. Clinical trials

    Azafaros registers open-label extension trial of nizubaglustat in rare lysosomal disorders

    The study, NCT07885917, is an open-label roll-over trial to evaluate long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, and Gaucher Disease Type 3. It plans to enroll 218 participants, with a start date of December 10, 2026, and primary completion by April 1, 2030.