Page 45 - Read Online
P. 45

Page 12 of 12            Politei et al. Rare Dis Orphan Drugs J 2024;3:10  https://dx.doi.org/10.20517/rdodj.2023.46

                   correlations from the phase 3 FACETS trial. Orphanet J Rare Dis 2018;13:68.  DOI  PubMed  PMC
               48.      Shayman JA, Hinkovska-galcheva V, Shu L. Inhibitors of glucosylceramide synthase. In: Kabayama K, Inokuchi J, editors.
                   Glycolipids. New York: Springer; 2023. pp. 271-88.  DOI
               49.      Wanner C, Kimonis V, Politei J, et al. Understanding and modifying Fabry disease: rationale and design of a pivotal phase 3 study and
                   results from a patient-reported outcome validation study. Mol Genet Metab Rep 2022;31:100862.  DOI  PubMed  PMC
               50.      Deegan PB, Goker-Alpan O, Geberhiwot T, et al. Venglustat, an orally administered glucosylceramide synthase inhibitor: assessment
                   over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study. Mol Genet Metab
                   2023;138:106963.  DOI  PubMed  PMC
               51.      Burlina AP, Sims KB, Politei JM, et al. Early diagnosis of peripheral nervous system involvement in Fabry disease and treatment of
                   neuropathic pain: the report of an expert panel. BMC Neurol 2011;11:61.  DOI  PubMed  PMC
               52.      Ries M, Mengel E, Kutschke G, et al. Use of gabapentin to reduce chronic neuropathic pain in Fabry disease. J Inherit Metab Dis
                   2003;26:413-4.  DOI
               53.      Gugelmo G, Vitturi N, Francini-Pesenti F, et al. Gastrointestinal manifestations and low-fodmap protocol in a cohort of Fabry disease
                   adult patients. Nutrients 2023;15:658.  DOI  PubMed  PMC
               54.      Lenders M, Boutin M, Auray-Blais C, Brand E. Effects of orally delivered alpha-galactosidase A on gastrointestinal symptoms in
                   patients with Fabry disease. Gastroenterology 2020;159:1602-4.  DOI  PubMed
               55.      Monticelli M, Hay Mele B, Allocca M, et al. Curcumin Has beneficial effects on lysosomal alpha-galactosidase: potential implications
                   for the cure of Fabry disease. Int J Mol Sci 2023;24:1095.  DOI  PubMed  PMC
               56.      Bertoldi G, Carraro G, Ravarotto V, et al. The effect of green tea as an adjuvant to enzyme replacement therapy on oxidative stress in
                   fabry disease: a pilot study. Front Nutr 2022;9:924710.  DOI  PubMed  PMC
   40   41   42   43   44   45   46   47   48   49   50