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Mucopolysaccharidosis III

  • Study on the Safety and Effectiveness of Rebisufligene Etisparvovec for Patients with Mucopolysaccharidosis Type IIIA

    Recruiting

    2 1 1
    Investigated Drugs:
    • Rebisufligene Etisparvovec
    Spain

  • Long-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA

    Recruiting

    3 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis III
    Investigated Drugs:
    • Rebisufligene Etisparvovec
    Spain

  • Study on the Safety and Initial Effects of AAV9-CAG-coh-SGSH in Children with Sanfilippo A Syndrome

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis III
    Investigated Drugs:
    • Adeno-Associated Viral Vector Serotype 9 Containing The Human Sulfamidase Gene
    Spain

  • Study of Weekly Infusions of JR-441 for Patients with Mucopolysaccharidosis Type IIIA

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis III
    Investigated Drugs:
    • N-Sulfoglucosamine Sulfohydrolase Fused To A Humanised Monoclonal Antibody Targeting Human Transferrin Receptor
    Germany

Investigational Drugs in This Location

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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.

Explore the full ecosystem of clinical research in Europe.

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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