| Naslov: | Eculizumab for thrombotic microangiopathy induced by onasemnogene abeparvovec in spinal muscular atrophy |
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| Avtorji: | ID Kersnik-Levart, Tanja (Avtor)
ID Olas Kar, Nina (Avtor)
ID Močnik Pegan, Chiara (Avtor)
ID Vrščaj, Eva (Avtor)
ID Troha Gergeli, Anja (Avtor)
ID Loboda, Tanja (Avtor)
ID Osredkar, Damjan (Avtor) |
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| Datoteke: |  PDF - Predstavitvena datoteka, prenos (358,58 KB)
MD5: 1E442385946E46AD0B54A491FC70E3CD
 URL - Izvorni URL, za dostop obiščite https://doi.org/10.1159/000546114
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| Jezik: | Angleški jezik |
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| Tipologija: | 1.03 - Drugi znanstveni članki |
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| Organizacija: |  UKC LJ - Univerzitetni klinični center Ljubljana
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| Povzetek: | 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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| Ključne besede: | spinal muscular atrophy, gene replacement therapy, onasemnogene abeparvovec, thrombotic microangiopathy, eculizumab |
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| Status publikacije: | Objavljeno |
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| Verzija publikacije: | Objavljena publikacija |
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| Leto izida: | 2025 |
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| Št. strani: | str. 133-140 |
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| Številčenje: | Vol. 15, issue 1 |
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| PID: | 20.500.12556/DiRROS-28102  |
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| UDK: | 616.8 |
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| ISSN pri članku: | 2296-9705 |
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| DOI: | 10.1159/000546114 |
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| COBISS.SI-ID: | 240351491 |
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| Opomba: | Nasl. z nasl. zaslona;
Opis vira z dne 24. 6. 2025;
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| Datum objave v DiRROS: | 10.03.2026 |
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| Število ogledov: | 69 |
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| Število prenosov: | 50 |
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| Metapodatki: |    |
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