Pegunigalsidase Alfa in Fabry Disease Patients with Severe Infusion Reactions to Other Enzyme Replacement Therapy: A Spanish Multi-Center Experience (ERA 2026) - Apr 13, 2026 - Abstract #3728; Pres time: Jun 4, 2026; 10:00 AM - 11:00 AM; Location: Research Zone; "This study aimed to evaluate the safety, tolerability, and clinical stability of PA in FD patients who previously discontinued agalsidase alfa or beta due to severe tolerability issues. Pegunigalsidase alfa represents a safe and effective "rescue" strategy for Fabry patients with history of severe IRRs. The PEGylation of the enzyme appears to successfully mask immunogenic epitopes, allowing for the continuation of essential therapy without compromising safety or biochemical control. These real-world data support PA as a viable alternative to overcome the limitations of other ERT in patients suffering severe infusion reactions"
Clinical • Fabry Disease • Genetic Disorders
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Laura Martinez Valenzuela 1; Monica Lopez 2; Carles Diez 1; Francisco Gomez Preciado 1; Juliana Bordignon Draibe 1; Silvia Vega-Gonzalez 1; Xavier Fulladosa Oliveras 1; Joan Torras Ambros1
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