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Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age.
Parini R, Pintos-Morell G, Hennermann JB, Hsu TR, Karabul N, Kalampoki V, Gurevich A, Ramaswami U; FOS Study Group. Parini R, et al. Drug Des Devel Ther. 2020 Jun 3;14:2149-2158. doi: 10.2147/DDDT.S249433. eCollection 2020. Drug Des Devel Ther. 2020. PMID: 32581513 Free PMC article.
Measuring patient experiences in Fabry disease: validation of the Fabry-specific Pediatric Health and Pain Questionnaire (FPHPQ).
Ramaswami U, Stull DE, Parini R, Pintos-Morell G, Whybra C, Kalkum G, Rohrbach M, Raluy-Callado M, Beck M, Chen WH, Wiklund I; FOS Investigators. Ramaswami U, et al. Among authors: parini r. Health Qual Life Outcomes. 2012 Sep 20;10:116. doi: 10.1186/1477-7525-10-116. Health Qual Life Outcomes. 2012. PMID: 22992222 Free PMC article.
Therapeutic goals in the treatment of Fabry disease.
Mehta A, West ML, Pintos-Morell G, Reisin R, Nicholls K, Figuera LE, Parini R, Carvalho LR, Kampmann C, Pastores GM, Lidove O. Mehta A, et al. Among authors: parini r. Genet Med. 2010 Nov;12(11):713-20. doi: 10.1097/GIM.0b013e3181f6e676. Genet Med. 2010. PMID: 20975569 Free article.
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document.
Biegstraaten M, Arngrímsson R, Barbey F, Boks L, Cecchi F, Deegan PB, Feldt-Rasmussen U, Geberhiwot T, Germain DP, Hendriksz C, Hughes DA, Kantola I, Karabul N, Lavery C, Linthorst GE, Mehta A, van de Mheen E, Oliveira JP, Parini R, Ramaswami U, Rudnicki M, Serra A, Sommer C, Sunder-Plassmann G, Svarstad E, Sweeb A, Terryn W, Tylki-Szymanska A, Tøndel C, Vujkovac B, Weidemann F, Wijburg FA, Woolfson P, Hollak CE. Biegstraaten M, et al. Among authors: parini r. Orphanet J Rare Dis. 2015 Mar 27;10:36. doi: 10.1186/s13023-015-0253-6. Orphanet J Rare Dis. 2015. PMID: 25885911 Free PMC article.
198 results