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6. The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease - A systematic literature review by a European panel of experts. Spada M; Baron R; Elliott PM; Falissard B; Hilz MJ; Monserrat L; Tøndel C; Tylki-Szymańska A; Wanner C; Germain DP Mol Genet Metab; 2019 Mar; 126(3):212-223. PubMed ID: 29785937 [TBL] [Abstract][Full Text] [Related]
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13. Genetics and Gene Therapy of Anderson-Fabry Disease. Simonetta I; Tuttolomondo A; Di Chiara T; Miceli S; Vogiatzis D; Corpora F; Pinto A Curr Gene Ther; 2018; 18(2):96-106. PubMed ID: 29618309 [TBL] [Abstract][Full Text] [Related]
14. Long-term enzyme replacement therapy for Fabry disease: efficacy and unmet needs in cardiac and renal outcomes. Kim JH; Lee BH; Hyang Cho J; Kang E; Choi JH; Kim GH; Yoo HW J Hum Genet; 2016 Nov; 61(11):923-929. PubMed ID: 27334365 [TBL] [Abstract][Full Text] [Related]
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16. Long-term enzyme replacement therapy is associated with reduced proteinuria and preserved proximal tubular function in women with Fabry disease. Prabakaran T; Birn H; Bibby BM; Regeniter A; Sørensen SS; Feldt-Rasmussen U; Nielsen R; Christensen EI Nephrol Dial Transplant; 2014 Mar; 29(3):619-25. PubMed ID: 24215016 [TBL] [Abstract][Full Text] [Related]
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20. [The neurological manifestations of Fabry disease. A review]. Firsov KV; Kotov AS Zh Nevrol Psikhiatr Im S S Korsakova; 2016; 116(9):98-105. PubMed ID: 27735906 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]