Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Gaillard, Jonathan; Gu, Andrew Ran; Neil Knierbein, Erin E.

doi:10.1016/j.jpeds.2023.113493

Cited by 6 publications

(4 citation statements)

References 24 publications

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“…Fatal cases of thrombotic microangiopathy and acute liver failure have been reported, 119 120 as well as potentially fatal conditions such as hemophagocytic syndrome and necrotizing enterocolitis. 121 122 …”

Section: Resultsmentioning

confidence: 99%

“…Fatal cases of thrombotic microangiopathy and acute liver failure have been reported, 119,120 as well as potentially fatal conditions such as hemophagocytic syndrome and necrotizing enterocolitis. 121,122 AAV9 titer measurement AAV9 is used as a vector to deliver the therapeutic gene in onasemnogene abeparvovec. However, if a patient has preexisting antibodies against AAV9 in their blood, these antibodies can neutralize the viral vector, preventing it from effectively delivering the therapeutic gene to the target cells.…”

Section: Safety Of Gene Replacement Therapy With Onasemnogene Abeparv...mentioning

confidence: 99%

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Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy

Zanoteli,

Araujo,

Becker

et al. 2024

Arq Neuropsiquiatr

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Spinal muscular atrophy linked to chromosome 5 (SMA-5q) is an autosomal recessive genetic disease caused by mutations in the SMN1. SMA-5q is characterized by progressive degeneration of the spinal cord and bulbar motor neurons, causing severe motor and respiratory impairment with reduced survival, especially in its more severe clinical forms. In recent years, highly effective disease-modifying therapies have emerged, either acting by regulating the splicing of exon 7 of the SMN2 gene or adding a copy of the SMN1 gene through gene therapy, providing a drastic change in the natural history of the disease. In this way, developing therapeutic guides and expert consensus becomes essential to direct the use of these therapies in clinical practice. This consensus, prepared by Brazilian experts, aimed to review the main available disease-modifying therapies, critically analyze the results of clinical studies, and provide recommendations for their use in clinical practice for patients with SMA-5q. This consensus also addresses aspects related to diagnosis, genetic counseling, and follow-up of patients under drug treatment. Thus, this consensus provides valuable information regarding the current management of SMA-5q, helping therapeutic decisions in clinical practice and promoting additional gains in outcomes.

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Section: Resultsmentioning

confidence: 99%

Section: Safety Of Gene Replacement Therapy With Onasemnogene Abeparv...mentioning

confidence: 99%

Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy

Zanoteli,

Araujo,

Becker

et al. 2024

Arq Neuropsiquiatr

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“…Fatal cases of thrombotic microangiopathy and acute liver failure have been reported, 60,61 as well as potentially fatal conditions such as hemophagocytic syndrome, 62 and necrotizing enterocolitis. 63 The STRONG study (NCT03381729) is a clinical trial that evaluated the safety and efficacy of an intrathecal single dose of onasemnogene abeparvovec in non-ambulatory patients with SMA who have three copies of the SMN2 and are aged 6 to under 60 months. 64 In the younger group (6 to under 24 months) treated with the medium dose, one out of thirteen patients (7.7%) achieved independent standing.…”

Section: Sod1-alsmentioning

confidence: 99%

“…Fatal cases of thrombotic microangiopathy and acute liver failure have been reported, 60 61 as well as potentially fatal conditions such as hemophagocytic syndrome, 62 and necrotizing enterocolitis. 63 …”

Section: Spinal Muscular Atrophy Linked To 5q (Sma-5q)mentioning

confidence: 99%

Gene-based therapies for neuromuscular disorders

Zanoteli,

França,

Marques

2024

Arq Neuropsiquiatr

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Neuromuscular diseases (NMD) include a broad group of medical conditions with both acquired and genetic causes. In recent years, important advances have been made in the treatment of genetically caused NMD, and most of these advances are due to the implementation of therapies aimed at gene regulation. Among these therapies, gene replacement, small interfering RNA (siRNA), and antisense antinucleotides are the most promising approaches. More importantly, some of these therapies have already gained regulatory approval or are in the final stages of approval. The review focuses on motor neuron diseases, neuropathies, and Duchenne muscular dystrophy, summarizing the most recent developments in gene-based therapies for these conditions.

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Onasemnogene-abeparvovec

2023

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Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Cited by 6 publications

References 24 publications

Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy

Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy

Gene-based therapies for neuromuscular disorders

Onasemnogene-abeparvovec

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