Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
Author:
Publisher
Elsevier BV
Subject
Pediatrics, Perinatology and Child Health
Reference25 articles.
1. Spinal muscular atrophy;Nicolau;Semin Pediatr Neurol,2021
2. Emerging therapies and challenges in spinal muscular atrophy;Farrar;Ann Neurol,2017
3. Severe Toxicity in Nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN;Hinderer;Hum Gene Ther,2018
4. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy;Chand;J Hepatol,2021
5. Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series;Chand;J Pediatr,2021
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1. Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’;Journal of Neuromuscular Diseases;2024-09-03
2. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders;Molecular Therapy;2024-07
3. Gene-based therapies for neuromuscular disorders;Arquivos de Neuro-Psiquiatria;2024-02-07
4. Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy;Arquivos de Neuro-Psiquiatria;2024-01
5. Onasemnogene-abeparvovec;Reactions Weekly;2023-11-25
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