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http://dx.doi.org/10.1002/jimd.70080 | DOI Listing |
J Inherit Metab Dis
September 2025
Department of Public Health, Chair of Nephrology, University Federico II of Naples, Naples, Italy.
J Clin Med
July 2025
Global Evidence and Outcomes, Data and Quantitative Sciences Institute, Takeda Development Center Americas, Inc., Cambridge, MA 02142, USA.
This systematic literature review aimed to identify studies assessing the clinical efficacy and real-world effectiveness of current and emerging treatments for Fabry disease. Searches of the MEDLINE, EMBASE, and Cochrane library databases, as well as relevant congress proceedings, were conducted to identify publications reporting on studies in patients of any age, sex, race, or ethnicity who received any approved or experimental treatment for Fabry disease, published before 17 June 2024. Of 1881 publications screened, 234 reported data on renal, cardiac, cerebrovascular, and disease severity outcomes from 225 studies.
View Article and Find Full Text PDFAdv Ther
February 2025
Cardiovascular Department, ASL8 Arezzo San Donato Hospital, Via Pietro Nenni 20, 52100, Arezzo, Italy.
Adv Ther
February 2025
Cytel, Inc., Waltham, MA, USA.
J Inherit Metab Dis
January 2025
University Hospital Würzburg, Würzburg, Germany.
Fabry disease is a progressive, X-linked lysosomal disorder caused by reduced or absent α-galactosidase A activity due to GLA variants. The effects of migalastat were examined in a cohort of 125 Fabry patients with migalastat-amenable GLA variants in the followME Pathfinders registry (EUPAS20599), an ongoing, prospective, patient-focused registry evaluating outcomes for current Fabry disease treatments. We report annualised estimated glomerular filtration rate (eGFR) and Fabry-associated clinical events (FACEs) in a cohort of patients who had received ≥3 years of migalastat treatment in a real-world setting.
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