| Title: | Eculizumab for thrombotic microangiopathy induced by onasemnogene abeparvovec in spinal muscular atrophy |
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| Authors: | ID Kersnik-Levart, Tanja (Author)
ID Olas Kar, Nina (Author)
ID Močnik Pegan, Chiara (Author)
ID Vrščaj, Eva (Author)
ID Troha Gergeli, Anja (Author)
ID Loboda, Tanja (Author)
ID Osredkar, Damjan (Author) |
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| Files: |  PDF - Presentation file, download (358,58 KB)
MD5: 1E442385946E46AD0B54A491FC70E3CD
 URL - Source URL, visit https://doi.org/10.1159/000546114
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| Language: | English |
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| Typology: | 1.03 - Other scientific articles |
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| Organization: |  UKC LJ - Ljubljana University Medical Centre
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| Abstract: | Introduction: Onasemnogene abeparvovec is one of the three disease-modifying therapies available that can significantly improve the outcome of patients with 5q-spinal muscular atrophy. Therapy-induced thrombotic microangiopathy is an ultra-rare, but potentially life-threatening condition of not yet clearly defined aetiology. Case Presentation: A case of a 2-year-old patient with 5q-spinal muscular atrophy, who developed thrombotic microangiopathy after gene replacement therapy with onasemnogene abeparvovec, is described. This severe adverse event was promptly recognized and successfully treated with the complement C5 inhibitor. Conclusion: Thrombotic microangiopathy is an ultra-rare, but potentially life-threatening condition that can occur after onasemnogene abeparvovec therapy. Anticipation of these serious adverse events, its prompt recognition and treatment is crucial for a better outcome. |
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| Keywords: | spinal muscular atrophy, gene replacement therapy, onasemnogene abeparvovec, thrombotic microangiopathy, eculizumab |
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| Publication status: | Published |
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| Publication version: | Version of Record |
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| Year of publishing: | 2025 |
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| Number of pages: | str. 133-140 |
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| Numbering: | Vol. 15, issue 1 |
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| PID: | 20.500.12556/DiRROS-28102  |
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| UDC: | 616.8 |
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| ISSN on article: | 2296-9705 |
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| DOI: | 10.1159/000546114 |
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| COBISS.SI-ID: | 240351491 |
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| Note: | Nasl. z nasl. zaslona;
Opis vira z dne 24. 6. 2025;
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| Publication date in DiRROS: | 10.03.2026 |
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| Views: | 70 |
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| Downloads: | 52 |
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