20.500.12556/DiRROS-28102 Eculizumab for thrombotic microangiopathy induced by onasemnogene abeparvovec in spinal muscular atrophy 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. spinal muscular atrophy gene replacement therapy onasemnogene abeparvovec thrombotic microangiopathy eculizumab spinalna mišična atrofija trombotična mikroangiopatija gensko zdravljenje true false true Angleški jezik Slovenski jezik Neznano 2026-03-10 10:50:06 2026-03-10 10:50:06 2026-03-11 03:59:14 0000-00-00 00:00:00 2025 0 0 Nasl. z nasl. zaslona; Opis vira z dne 24. 6. 2025; str. 133-140 issue 1 Vol. 15 2025 0000-00-00 Zaloznikova Objavljeno NiDoloceno 0000-00-00 0000-00-00 0000-00-00 616.8 2296-9705 10.1159/000546114 240351491 RAZ_Kersnik-Levart_Tanja_2025.pdf RAZ_Kersnik-Levart_Tanja_2025.pdf 1 1E442385946E46AD0B54A491FC70E3CD 44a29da3ca057f5174391207f1348b9fea37b0255b2d67b48c88738970d4a495 b52fdb96-1c66-11f1-acf8-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=41528 https://doi.org/10.1159/000546114 0 bfa06d20-1c66-11f1-acf8-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=41529 Univerzitetni klinični center Ljubljana 0 0 0