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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Effectiveness of Dexamethasone in Preventing Prenatal Virilization in Female Patients with Congenital Adrenal Hyperplasia

    Recruiting

    2 1 1
    Investigated Diseases:
    • Adrenogenital syndrome
    Investigated Drugs:
    • Dexamethasone
    Germany

  • Study on C1 Esterase Inhibitor for Treating and Preventing Hereditary Angioedema Attacks in Patients with C1-INH Deficiency

    Recruiting

    3 1 1
    Investigated Drugs:
    • C1 Esterase Inhibitor (Human)
    • Sodium Chloride
    Bulgaria Romania

  • 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 Long-Term Safety of Idursulfase-IT and Elaprase for Patients with Hunter Syndrome and Cognitive Impairment

    Recruiting

    3 1 1
    Investigated Diseases:
    • Mucopolysaccharidosis II
    Investigated Drugs:
    • Idursulfase
    France

  • Oxygen Therapy for Pregnant Women to Support Heart Development in Unborn Babies with Underdeveloped Left Heart Structures

    Recruiting

    3 1 1 1
    Investigated Drugs:
    • Oxygen
    Germany
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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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