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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on the Long-Term Safety of Alpelisib for Patients with PIK3CA-Related Overgrowth Spectrum (PROS)

    Not recruiting

    2 1 1
    Investigated Drugs:
    • Alpelisib
    France Ireland Spain

  • Study on Long-Term Safety and Effects of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Trials

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Spinal muscular atrophy
    Investigated Drugs:
    • Apitegromab
    Belgium France Germany Italy The Netherlands Poland +1

  • Study on ALXN1850 for Adolescents and Adults with Hypophosphatasia Not Previously Treated with Asfotase Alfa

    Not recruiting

    3 1
    Investigated Diseases:
    • Hypophosphatasia
    Investigated Drugs:
    • Alxn1850
    Austria Belgium France Germany Italy Poland +2

  • Study on Salbutamol for Improving Muscle Strength in Patients with Congenital Myopathy

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Congenital myopathy
    Investigated Drugs:
    • Salbutamol
    Sweden

  • Study on Patidegib Gel for Reducing Basal Cell Carcinomas in Adults with Gorlin Syndrome

    Not recruiting

    3 1
    Investigated Drugs:
    • Patidegib Hydrochloride Isopropanolate
    France Germany Italy The Netherlands Poland Spain

  • Study on Long-Term Safety of Donidalorsen for Patients with Hereditary Angioedema

    Not recruiting

    3 1 1
    Investigated Drugs:
    • Donidalorsen
    Belgium Bulgaria France Germany Italy The Netherlands +2
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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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