First Known Case of Rare Immunological Syndrome After SMA Gene Therapy Reported - AJMC.com Managed Markets Network

The case describes hemophagocytic lymphohistiocytosis (HLH), which occurred in a young boy in Italy with spinal muscular atrophy, who was treated with onasemnogene abeparvovec.

Researchers in Italy published what they said is the first case of a rare and life-threatening syndrome characterized by excessive immune activation following gene replacement therapy for spinal musc… [+3863 chars]

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