Amicus Therapeutics(US:FOLD) Globenewswire·2026-02-03 13:00Core Insights - Amicus Therapeutics presented new data on Galafold and POMBILITI + OPFOLDA at the 22nd Annual WORLDSymposium 2026, highlighting advancements in the treatment of Fabry disease and late-onset Pompe disease [1][2] Fabry Disease - Galafold (migalastat) is an oral pharmacological chaperone for treating Fabry disease in adults with amenable GLA variants, stabilizing dysfunctional enzymes to clear disease substrates [8] - Approximately 35 to 50 percent of individuals with Fabry disease may have amenable GLA variants, with variability based on geography [8] - Oral presentations include real-world effectiveness of migalastat versus enzyme replacement therapy in treatment-naïve patients and long-term outcomes from the followME Pathfinders registry [3][4] Pompe Disease - POMBILITI + OPFOLDA is a two-component therapy for late-onset Pompe disease, combining cipaglucosidase alfa and miglustat to enhance enzyme uptake and activity [12] - The therapy is indicated for adult patients weighing ≥40 kg who are not improving on current enzyme replacement therapy [13] - Presentations include 208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease, focusing on muscle function and pulmonary function [6][6] WORLDSymposium - WORLDSymposium serves as a platform for researchers, clinicians, and patient advocacy groups to share the latest discoveries related to lysosomal diseases [16] - The symposium features presentations on both basic science and clinical trials, emphasizing advancements in the understanding and treatment of lysosomal diseases [16] Company Overview - Amicus Therapeutics is a global biotechnology company focused on developing transformative medicines for rare diseases, with a strong commitment to patient care [17]