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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Study on Luspatercept for Patients with Lower-risk Myelodysplastic Syndrome Requiring Red Blood Cell Transfusions

    Not recruiting

    3 1 1 1
    Investigated Drugs:
    • Luspatercept
    Belgium Czechia France Germany Italy Poland +1

  • Study of Afamelanotide for Patients with Erythropoietic Protoporphyria (EPP)

    Not recruiting

    1 1 1 1
    Investigated Diseases:
    • Porphyria non-acute
    Investigated Drugs:
    • Afamelanotide
    Belgium The Netherlands

  • Study on the Safety and Effects of Acetylcysteine Amide for Patients Aged 12 and Over with Hereditary Cystatin C Amyloid Angiopathy

    Not recruiting

    2 1 1
    Investigated Diseases:
    • Cerebral amyloid angiopathy
    Investigated Drugs:
    • Acetylcysteine Amide
    Iceland

  • Study on Aficamten for Adults with Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

    Not recruiting

    3 1
    Investigated Diseases:
    • Hypertrophic cardiomyopathy
    Investigated Drugs:
    • Aficamten
    Denmark France Germany Greece Hungary Iceland +5

  • Study on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency

    Not recruiting

    3 1 1
    Investigated Diseases:
    • Ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency
    Investigated Drugs:
    • Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1
    France Spain

  • Study on the Safety and Effects of INZ-701 for Infants with ENPP1 or ABCC6 Deficiency

    Not recruiting

    1 1 1
    Investigated Diseases:
    • Ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency
    • Arteriosclerosis
    Investigated Drugs:
    • Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1
    Spain
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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.

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