ArticleMolecular genetics and metabolism reports2025
Successful desensitization protocol to alglucosidase and avalglucosidase alfa in a patient with infantile-onset Pompe disease.
Article in Molecular genetics and metabolism reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
2 citing papers in PubMed.
- Combined omalizumab and desensitization to control IgE-mediated hypersensitivity in enzyme replacement therapy for late-onset Pompe disease.Orphanet journal of rare diseases · 2026Article
- Management of hypersensitivity in a patient with late-onset Pompe disease experiencing recurrent infusion-related reactions.Journal of neurology · 2025Article
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Infantile-onset Pompe disease is a lysosomal disease characterized by cardiomyopathy and muscle weakness that, without specific treatment, is fatal within the first two years of life. We present the case of an infant who developed anaphylaxia to enzyme replacement therapy with alglucosidase-alfa. We provide a desensitization protocol to alglucosidase-alfa and, for the first time, a desensitization protocol to avalglucosidase-alfa, both delivered in a reasonable time of <6 h, and without any further reactions in the patient.
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Registered trials
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