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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Hereditary neuropathic amyloidosis
    Investigated Drugs:
    • Single Guide Rna Targeting The Human Ttr Gene
    • Ziclumeran
    France Sweden

  • Study on the Safety of Vonafexor for Patients with Alport Syndrome at Risk of Progression

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Alport’s syndrome
    Investigated Drugs:
    • Vonafexor
    France Germany Spain

  • Study on the Safety and Effectiveness of Crofelemer for Children with Microvillus Inclusion Disease (MVID)

    Not recruiting

    4 1
    Investigated Diseases:
    • Microvillous inclusion disease
    Investigated Drugs:
    • Crofelemer
    Italy

  • Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells

    Not recruiting

    2 1 1 1
    Investigated Drugs:
    • Exagamglogene Autotemcel
    • Plerixafor
    • Busulfan
    Belgium Italy

  • Study on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies

    Not recruiting

    1 1 1 1
    Investigated Drugs:
    • Imlifidase
    • Delandistrogene Moxeparvovec
    Spain

  • Study on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Osteogenesis imperfecta
    Investigated Drugs:
    • Allogeneic Fetal Mesenchymal Stem Cells
    The Netherlands Sweden
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European Clinical Trials Information Network

The European Clinical Trials Information Network is transforming access to clinical trial information for patients across Europe. By overcoming language barriers, our platform allows everyone, regardless of their native language, to easily find relevant and comprehensible clinical trial data. This innovative service connects patients to cutting-edge treatments, creating a unified European community where health and progress transcend borders.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic centres, national regulators, and commercial sponsors. We aim to keep all content accurate and up to date.

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The information provided on this website is mainly derived from international public clinical trial databases (CTIS.eu). In addition, we are including supplementary sources, such as information provided by academic centers and national regulators, as well as information supplied by commercial sponsors (pharmaceutical companies and contract research organizations). We try to ensure that the information provided is accurate and up to date.

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