Endocrinology Trials

  • 09/03/2016


    A non-interventional Post Authorization Safety Study (PASS) to evaluate long-term safety of Orfadin treatment in hypertyrosinemia type 1 (HT-1) patients in standard clinical care

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  • 01/05/2022

    Fabry BALANCE

    A Randomized Double blind Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta

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  • 05/08/2030

    Predictive measures to stratify clinical outcomes in children and adults with Gaucher disease and responses to specific therapies

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  • 31/12/2021

    Fabry BRIDGE

    An Open Label Study of the Safety and Efficacy of PRX-102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

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  • 01/12/2028

    Niemann-Pick Disease registry

    An EU rare diseases registry for Niemann-Pick Disease type A B and C

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  • 23/11/2013

    Long Term Retrospective Growth of Children With Phenylketonuria

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  • 24/01/2021

    MPS VI Clinical Surveillance Program (CSP)

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  • 31/12/2013

    EURO-WABB: An EU Rare Disease Registry for Wolfram Syndrome Alström Syndrome Bardet Biedl Syndrome and other Rare Diabetes Syndromes.

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  • 07/01/2012


    Fabry Outcome Survey (FOS)

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  • 31/12/2019


    A Phase 1/2 Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency

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