UKM Fabry-Zentrum


  1. Pogoda C, Brand SM, Duning T, Schmidt-Pogoda A, Sindermann J, Lenders M, Brand E. Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease. Front Cardiovasc Med. 2023;10:1223635. 
  2. Lenders M, Feidicker LM, Brand SM, Brand E. Characterization of pre-existing anti-PEG and anti-AGAL antibodies towards PRX-102 in patients with Fabry disease. Front Immunol. 2023;14:1266082. 
  3. Burlina A, Brand E, Hughes D, Kantola I, Krӓmer J, Nowak A, Tøndel C, Wanner C, Spada M. An expert consensus on the recommendations for the use of biomarkers in Fabry disease. Mol Genet Metab. 2023;139:107585. 
  4. Müntze J, Lau K, Cybulla M, Brand E, Cairns T, Lorenz L, Üçeyler N, Sommer C, Wanner C, Nordbeck P. Patient reported quality of life and medication adherence in Fabry disease patients treated with migalastat: A prospective, multicenter study. Mol Genet Metab. 2023;138:106981.
  5. Winter Y, Hilz M, Beuschlein F, Tsukimura T, Seifritz E, Lenders M, Brand E, Hennermann JB, Nowak A. Screening for health-related quality of life and its determinants in Fabry disease: A cross-sectional multicenter study. Mol Genet Metab. 2023;140:107692.
  6. Hopkin RJ, Cabrera GH, Jefferies JL, Yang M, Ponce E, Brand E, Feldt-Rasmussen U, Germain DP, Guffon N, Jovanovic A, Kantola I, Karaa A, Martins AM, Tøndel C, Wilcox WR, Yoo HW, Burlina AP, Mauer M. Clinical outcomes among young patients with Fabry disease who initiated agalsidase beta treatment before 30 years of age: An analysis from the Fabry Registry. Mol Genet Metab. 2023;138:106967
  7. Lenders M, Brand E. Assessment and impact of dose escalation on anti-drug antibodies in Fabry disease. Front Immunol. 2022;13:1024963.
  8. Lenders M, Pollmann S, Terlinden M, Brand E. Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa. Mol Ther Methods Clin Dev. 2022;26:323-330.
  9. Lenders M, Nordbeck P, Kurschat C, Eveslage M, Karabul N, Kaufeld J, Hennermann JB, Patten M, Cybulla M, Müntze J, Üçeyler N, Liu D, Das AM, Sommer C, Pogoda C, Reiermann S, Duning T, Gaedeke J, von Cossel K, Blaschke D, Brand SM, Mann WA, Kampmann C, Muschol N, Canaan-Kühl S, Brand E. Treatment of Fabry Disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS). Eur Heart J Cardiovasc Pharmacother. 2022;8:272-281.
  10. Lenders M, Brand E. Fabry disease - a multisystemic disease with gastrointestinal manifestations. Gut Microbes. 2022;14:2027852
  11. Pollmann S, Scharnetzki D, Manikowski D, Lenders M, Brand E. Endothelial Dysfunction in Fabry Disease Is Related to Glycocalyx Degradation. Front Immunol. 2021;12:789142.
  12. Jehn U, Bayraktar S, Pollmann S, Van Marck V, Weide T, Pavenstädt H, Brand E, Lenders M. α-Galactosidase a Deficiency in Fabry Disease Leads to Extensive Dysregulated Cellular Signaling Pathways in Human Podocytes. Int J Mol Sci. 2021;22:11339.
  13. Lenders M, Brand E. Mechanisms of Neutralizing Anti-drug Antibody Formation and Clinical Relevance on Therapeutic Efficacy of Enzyme Replacement Therapies in Fabry Disease. Drugs. 2021;81:1969-1981.
  14. Lenders M, Brand E. Precision medicine in Fabry disease. Nephrol Dial Transplant. 2021;36:14-23. 
  15. Lenders M, Scharnetzki D, Heidari A, Di Iorio D, Wegner SV, Brand E. Generation and Characterization of a Polyclonal Human Reference Antibody to Measure Anti-Drug Antibody Titers in Patients with Fabry Disease. Int J Mol Sci. 2021;22:2680.
  16. Lenders M, Brand E. Fabry Disease: The Current Treatment Landscape. Drugs. 2021;81:635-645.
  17. Lenders M, Nordbeck P, Kurschat C, Karabul N, Kaufeld J, Hennermann JB, Patten M, Cybulla M, Müntze J, Üçeyler N, Liu D, Das AM, Sommer C, Pogoda C, Reiermann S, Duning T, Gaedeke J, Stumpfe K, Blaschke D, Brand SM, Mann WA, Kampmann C, Muschol N, Canaan-Kühl S, Brand E. Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS). Clin Pharmacol Ther. 2020;108:326-337.
  18. Lenders MBrand E. FAbry STabilization indEX (FASTEX): Clinical evaluation of disease progression in Fabry patients. Mol Genet Metab. 2020;129:142-149.
  19. 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;159:1602-1604.
  20. Lenders M, Nordbeck P, Canaan-Kühl S, Kreul L, Duning T, Lorenz L, Pogoda C, Brand SM, Wanner C, Brand E. Treatment switch in Fabry disease- a matter of dose? J Med Genet. 2020:jmedgenet-2020-106874.
  21. Stappers F, Scharnetzki D, Schmitz B, Manikowski D, Brand SM, Grobe K, Lenders MBrand E. Neutralising anti-drug antibodies in Fabry disease can inhibit endothelial enzyme uptake and activity. J Inherit Metab Dis. 2020;43:334-347.
  22. Lenders M, Stappers F, Brand E. In vitro and in vivo amenability to migalastat in Fabry disease. Mol Ther Methods Clin Dev. 2020;19:24-34.
  23. Scharnetzki D, Stappers F, Lenders MBrand E. Detailed epitope mapping of neutralizing anti-drug antibodies against recombinant α-galactosidase A in patients with Fabry disease. Mol Genet Metab. 2020;131:229-234.
  24. van der Veen SJ, Vlietstra WJ, van Dussen L, van Kuilenburg ABP, Dijkgraaf MGW, Lenders MBrand E, Wanner C, Hughes D, Elliott PM, Hollak CEM, Langeveld M. Predicting the Development of Anti-Drug Antibodies against Recombinant alpha-Galactosidase A in Male Patients with Classical Fabry Disease. Int J Mol Sci. 2020;21:5784.
  25. Krämer J, Glaser F, Hasselblatt M, Brand E, Pogoda C, Lenders M, Wiendl H, Meuth SG, Duning T. Case Report: A Spinal Ischemic Lesion in a 24-Year-Old Patient With Fabry Disease.  Front Immunol. 2020;11:595514.
  26. Wanner C, Feldt-Rasmussen U, Jovanovic A, Linhart A, Yang M, Ponce E, Brand E, Germain DP, Hughes DA, Jefferies JL, Martins AM, Nowak A, Vujkovac B, Weidemann F, West ML, Ortiz A. Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis. ESC Heart Fail. 2020;7:825-834.
  27. Xiao K, Lu D, Hoepfner J, Santer L, Gupta S, Pfanne A, Thum S, Lenders MBrand E, Nordbeck P, Thum T. Circulating microRNAs in Fabry Disease. Sci Rep. 2019;9:15277.
  28. Lenders M, Stappers F, Niemietz C, Schmitz B, Boutin M, Ballmaier PJ, Zibert A, Schmidt H, Brand SM, Auray-Blais C, Brand E. Mutation-specific Fabry disease patient-derived cell model to evaluate the amenability to chaperone therapy. J Med Genet. 2019;56:548-556.
  29. Lenders M, Neußer LP, Rudnicki M, Nordbeck P, Canaan-Kühl S, Nowak A, Cybulla M, Schmitz B, Lukas J, Wanner C, Brand SM, Brand E. Dose-Dependent Effect of Enzyme Replacement Therapy on Neutralizing Antidrug Antibody Titers and Clinical Outcome in Patients with Fabry Disease. J Am Soc Nephrol. 2018;29:2879-2889.
  30. Germain DP, Brand E, Burlina A, Cecchi F, Garman SC, Kempf J, Laney DA, Linhart A, Maródi L, Nicholls K, Ortiz A, Pieruzzi F, Shankar SP, Waldek S, Wanner C, Jovanovic A. Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study. Mol Genet Genomic Med. 2018;6:492-503.
  31. Lenders M, Schmitz B, Brand SM, Brand E. Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact? Orphanet J Rare Dis. 2018;13:171.
  32. Lenders MBrand E. Effects of Enzyme Replacement Therapy and Antidrug Antibodies in Patients with Fabry Disease. J Am Soc Nephrol. 2018;29:2265-2278.
  33. Lenders M, Schmitz B, Brand SM, Foell D, Brand E. Characterization of drug-neutralizing antibodies in patients with Fabry disease during infusion. J Allergy Clin Immunol. 2018;141:2289-2292.e7.
  34. Krämer J, Lenders M, Canaan-Kühl S, Nordbeck P, Üçeyler N, Blaschke D, Duning T, Reiermann S, Stypmann J, Brand SM, Gottschling T, Störk S, Wanner C, Sommer C, Brand E, Weidemann F. Fabry disease under enzyme replacement therapy-new insights in efficacy of different dosages. Nephrol Dial Transplant. 2018;33:1362-1372.
  35. Lenders M, Oder D, Nowak A, Canaan-Kühl S, Arash-Kaps L, Drechsler C, Schmitz B, Nordbeck P, Hennermann JB, Kampmann C, Reuter S, Brand SM, Wanner C, Brand E. Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients with Fabry disease. J Intern Med. 2017;282:241-253.
  36. Lenders M, Schmitz B, Stypmann J, Duning T, Brand SM, Kurschat C, Brand E. Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease. Nephrol Dial Transplant. 2017;32:2090-2097.
  37. Lenders M, Hennermann JB, Kurschat C, Rolfs A, Canaan-Kühl S, Sommer C, Üçeyler N, Kampmann C, Karabul N, Giese AK, Duning T, Stypmann J, Krämer J, Weidemann F, Brand SM, Wanner C, Brand E. Multicenter Female Fabry Study (MFFS) - clinical survey on current treatment of females with Fabry disease. Orphanet J Rare Dis. 2016;11:88.
  38. Lenders M, Weidemann F, Kurschat C, Canaan-Kühl S, Duning T, Stypmann J, Schmitz B, Reiermann S, Krämer J, Blaschke D, Wanner C, Brand SM, Brand E. Alpha-Galactosidase A p.A143T, a non-Fabry disease-causing variant. Orphanet J Rare Dis. 2016;11:54.
  39. Lidove O, Barbey F, Niu DM, Brand E, Nicholls K, Bizjajeva S, Hughes DA. Fabry in the older patient: Clinical consequences and possibilities for treatment. Mol Genet Metab. 2016;118:319-25.
  40. Schmitz B, Thorwesten L, Lenders M, Duning T, Stypmann J, Brand E, Brand SM. Physical Exercise in Patients with Fabry Disease - a Pilot Study. Int J Sports Med. 2016;37:1066-1072.
  41. Lenders M, Canaan-Kühl S, Krämer J, Duning T, Reiermann S, Sommer C, Stypmann J, Blaschke D, Üçeyler N, Hense HW, Brand SM, Wanner C, Weidemann F, Brand E. Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up. J Am Soc Nephrol. 2016;27:952-62.
  42. Lenders M, Stypmann J, Duning T, Schmitz B, Brand SM, Brand E. Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease. J Am Soc Nephrol. 2016;27:256-264.
  43. Lenders M, Karabul N, Duning T, Schmitz B, Schelleckes M, Mesters R, Hense HW, Beck M, Brand SM, Brand E. Thromboembolic events in Fabry disease and the impact of factor V Leiden. Neurology. 2015;84:1009-1016.
  44. Schelleckes M, Lenders M, Guske K, Schmitz B, Tanislav C, Ständer S, Metze D, Katona I, Weis J, Brand SM, Duning T, Brand E. Cryptogenic stroke and small fiber neuropathy of unknown etiology in patients with alpha-galactosidase A -10T genotype. Orphanet J Rare Dis. 2014;9:178.
  45. Weidemann F, Krämer J, Duning T, Lenders M, Canaan-Kühl S, Krebs A, Guerrero González H, Sommer C, Üçeyler N, Niemann M, Störk S, Schelleckes M, Reiermann S, Stypmann J, Brand SM, Wanner C, Brand E. Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch. J Am Soc Nephrol. 2014;25:837-849.
  46. Lenders M, Duning T, Schelleckes M, Schmitz B, Stander S, Rolfs A, Brand SM, Brand E. Multifocal white matter lesions associated with the D313Y mutation of the α-galactosidase A gene. PLoS One. 2013;8:e55565.
  47. Engelen MA, Brand E, Baumeister TB, Marquardt T, Duning T, Osada N, Schaefer RM, Stypmann J. Effects of enzyme replacement therapy in adult patients with Fabry disease on cardiac structure and function: a retrospective cohort study of the Fabry Munster Study (FaMuS) data. BMJ Open. 2012;2:bmjopen-2012-000879.