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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: kampmann c. Genet Med. 2010 Nov;12(11):713-20. doi: 10.1097/GIM.0b013e3181f6e676. Genet Med. 2010. PMID: 20975569 Free article.
Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis.
Ramaswami U, Beck M, Hughes D, Kampmann C, Botha J, Pintos-Morell G, West ML, Niu DM, Nicholls K, Giugliani R; FOS Study Group. Ramaswami U, et al. Among authors: kampmann c. Drug Des Devel Ther. 2019 Oct 25;13:3705-3715. doi: 10.2147/DDDT.S207856. eCollection 2019. Drug Des Devel Ther. 2019. PMID: 31749608 Free PMC article.
Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease.
Ries M, Clarke JT, Whybra C, Timmons M, Robinson C, Schlaggar BL, Pastores G, Lien YH, Kampmann C, Brady RO, Beck M, Schiffmann R. Ries M, et al. Among authors: kampmann c. Pediatrics. 2006 Sep;118(3):924-32. doi: 10.1542/peds.2005-2895. Pediatrics. 2006. PMID: 16950982 Clinical Trial.
Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry.
Beck M, Ramaswami U, Hernberg-Ståhl E, Hughes DA, Kampmann C, Mehta AB, Nicholls K, Niu DM, Pintos-Morell G, Reisin R, West ML, Schenk J, Anagnostopoulou C, Botha J, Giugliani R. Beck M, et al. Among authors: kampmann c. Orphanet J Rare Dis. 2022 Jun 20;17(1):238. doi: 10.1186/s13023-022-02392-9. Orphanet J Rare Dis. 2022. PMID: 35725623 Free PMC article. Review.
Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis.
Beck M, Hughes D, Kampmann C, Larroque S, Mehta A, Pintos-Morell G, Ramaswami U, West M, Wijatyk A, Giugliani R; Fabry Outcome Survey Study Group. Beck M, et al. Among authors: kampmann c. Mol Genet Metab Rep. 2015 Mar 5;3:21-7. doi: 10.1016/j.ymgmr.2015.02.002. eCollection 2015 Jun. Mol Genet Metab Rep. 2015. PMID: 26937390 Free PMC article.
151 results