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Spinal muscular atrophy

  • Study of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)

    Recruiting

    2 1 1
    Investigated Drugs:
    • Apitegromab
    Belgium France Italy The Netherlands Spain

  • Study on the Early Use of Risdiplam for Children with Spinal Muscular Atrophy After Gene Therapy

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Spinal muscular atrophy
    Investigated Drugs:
    • Risdiplam
    Germany Poland

  • Study on the Effectiveness and Safety of Risdiplam for Children with Spinal Muscular Atrophy After Gene Therapy

    Recruiting

    3 1 1 1
    Investigated Diseases:
    • Spinal muscular atrophy
    Investigated Drugs:
    • Risdiplam
    Germany Poland

  • Study of BIIB115 in patients aged 15‑60 years with spinal muscular atrophy who are treatment‑naïve or previously treated with risdiplam

    Not yet recruiting

    3 1 1
    Investigated Drugs:
    • BIIB115
    Finland France Germany Ireland Italy Poland +1

  • Study on the Safety and Tolerability of BIIB115 for Children with Spinal Muscular Atrophy Previously Treated with Gene Therapy

    Not recruiting

    1 1
    Investigated Diseases:
    • Spinal muscular atrophy
    Investigated Drugs:
    • BIIB115
    Belgium France Germany Italy The Netherlands Poland

  • Long-term Safety Study of Onasemnogene Abeparvovec for Patients with Spinal Muscular Atrophy

    Not recruiting

    3 1 1 1
    Investigated Diseases:
    • Spinal muscular atrophy
    Investigated Drugs:
    • Onasemnogene Abeparvovec
    Belgium Denmark France Italy The Netherlands Spain
1 2 3
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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.

Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).

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