Long-term safety and efficacy of pegunigalsidase alfa in treatment-naive Fabry disease patients in F60/BRILLIANCE trial (SSIEM 2026) - Aug 17, 2026 - Abstract #P-282; Pres time: Aug 26, 2026; 05:30 PM - 06:30 PM; Location: Hall 4&5; P3 | "PA treatment for ≤10 years in ERT-naive FD patients was well-tolerated and associated with stable renal function and sustained lyso-Gb3 level reduction. Although patients with BL eGFR>110mL/min/1.73m² had steeper declines than those with BL eGFR110 is warranted."
Clinical • CNS Disorders • Fabry Disease • Genetic Disorders • Migraine
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Ozlem Goker-Alpan 1; John A Bernat 2; Robert J Hopkin 5; Derralynn Hughes 4; Aleš Linhart 5; Antonio Pisani 6; Camilla Tøndel 7; William R Willcox 8; Irene Koulinska 9; David Lee 10; Irene Rastelletti 11; Eric L Wallace 12
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