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Article

Hemophagocytic lymphohistiocytosis following gene replacement therapy in a child with type 1 spinal muscular atrophy.

Journal of clinical pharmacy and therapeutics - 1 Sept 2022

Galletta Francesca, Cucinotta Ugo, Marseglia Lucia, Cacciola Annalisa, Gallizzi Romina, Cuzzocrea Salvatore, Messina Sonia, Toscano Antonio, Gitto Eloisa

Abstract excerpt

WHAT IS KNOWN AND OBJECTIVE: Onasemnogene abeparvovec (OA) is the first gene replacement therapy for the treatment of paediatric patients with bi-allelic mutations in the SMN1 gene. Efficacy and safety of OA have been assessed in several studies with promising results, despite rare side effects have been described. CASE SUMMARY: A 3-year-old child with spinal muscular atrophy was treated with OA and subsequently...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 35924856
DOI: 10.1111/jcpt.13733

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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