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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease

    Recruiting

    1 1 1
    Investigated Diseases:
    • Pelizaeus-Merzbacher disease
    Investigated Drugs:
    • 2′-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Plp1 Pre-Mrna
    France Germany The Netherlands

  • Study of Intranasal Sufentanil and Intravenous Morphine for Severe Sickle Cell Pain Crisis in Children

    Recruiting

    3 1 1
    Investigated Diseases:
    • Sickle cell anaemia with crisis
    Investigated Drugs:
    • Sufentanil
    • Morphine Hydrochloride
    • Sodium Chloride
    France

  • Study of ISIS 678354 for Patients with Familial Chylomicronemia Syndrome Previously Treated with Volanesorsen

    Recruiting

    3 1 1
    Investigated Diseases:
    • Familial hypertriglyceridaemia
    • Hyperchylomicronaemia
    Investigated Drugs:
    • Isis 678354 Sodium Salt
    Sweden

  • Study on the Long-term Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy

    Recruiting

    2 1 1
    Investigated Drugs:
    • Edg-5506
    Belgium Denmark France Germany Italy The Netherlands +1

  • Study on the Effects of Avalglucosidase Alfa Enzyme Replacement Therapy in Patients with Pompe Disease

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Glycogen storage disease type II
    Investigated Drugs:
    • Avalglucosidase Alfa
    Denmark

  • Study on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease

    Recruiting

    2 1 1 1
    Investigated Diseases:
    • Glycogen storage disease type II
    Investigated Drugs:
    • Adeno-Associated Virus Serotype 9 Vector Containing The Human Lamp2 Isoform B Transgene
    • Methylprednisolone Sodium Succinate
    • Eculizumab
    Germany Italy
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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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