Thrombotic microangiopathy following gene therapy for 5q-spinal muscular atrophy
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Onasemnogene abeparvovec (OA) is the first gene replacement therapy (GT) approved for 5q spinal muscular atrophy (SMA). While effective, it can cause severe side effects, including thrombotic microangiopathy (TMA). The pathophysiology, risk factors, and manage... ...
