Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1
Garrelfs, Sander F, Frishberg, Yaacov, Hulton, Sally A, Koren, Michael J, O’Riordan, William D, Cochat, Pierre, Deschênes, Georges, Shasha-Lavsky, Hadas, Saland, Jeffrey M, van’t Hoff, William G, Fuster, Daniel G, Magen, Daniella, Moochhala, Shabbir H, Schalk, Gesa, Simkova, Eva, Groothoff, Jaap W, Sas, David J, Meliambro, Kristin A, Lu, Jiandong, Sweetser, Marianne T, Garg, Pushkal P, Vaishnaw, Akshay K, Gansner, John M, McGregor, Tracy L, Lieske, John C
Published in The New England journal of medicine (01.04.2021)
Published in The New England journal of medicine (01.04.2021)
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Journal Article
Safety, pharmacodynamics, and exposure-response modeling results from a first-in-human phase 1 study of nedosiran (PHYOX1) in primary hyperoxaluria
Hoppe, Bernd, Koch, Annelize, Cochat, Pierre, Garrelfs, Sander F., Baum, Michelle A., Groothoff, Jaap W., Lipkin, Graham, Coenen, Martin, Schalk, Gesa, Amrite, Aniruddha, McDougall, David, Barrios, Kelly, Langman, Craig B.
Published in Kidney international (01.03.2022)
Published in Kidney international (01.03.2022)
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Journal Article
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. Reply
Garrelfs, Sander F, Gansner, John M, Lieske, John C
Published in The New England journal of medicine (11.11.2021)
Published in The New England journal of medicine (11.11.2021)
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Journal Article
Patients with primary hyperoxaluria type 2 have significant morbidity and require careful follow-up
Garrelfs, Sander F., Rumsby, Gill, Peters-Sengers, Hessel, Erger, Florian, Groothoff, Jaap W., Beck, Bodo B., Oosterveld, Michiel J.S., Pelle, Alessandra, Neuhaus, Thomas, Adams, Brigitte, Cochat, Pierre, Salido, Eduardo, Lipkin, Graham W., Hoppe, Bernd, Hulton, Sally-Anne
Published in Kidney international (01.12.2019)
Published in Kidney international (01.12.2019)
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Journal Article
A report from the European Hyperoxaluria Consortium (OxalEurope) Registry on a large cohort of patients with primary hyperoxaluria type 3
Martin-Higueras, Cristina, Garrelfs, Sander F., Groothoff, Jaap W., Jacob, Dorrit E., Moochhala, Shabbir H., Bacchetta, Justine, Acquaviva, Cecile, Zaniew, Marcin, Sikora, Przymyslaw, Beck, Bodo B., Hoppe, Bernd
Published in Kidney international (01.09.2021)
Published in Kidney international (01.09.2021)
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Journal Article
Glyoxylate reductase: Definitive identification in human liver mitochondria, its importance for the compartment‐specific detoxification of glyoxylate
Garrelfs, Sander F., Chornyi, Serhii, Brinke, Heleen, Ruiter, Jos, Groothoff, Jaap, Wanders, Ronald J. A.
Published in Journal of inherited metabolic disease (01.03.2024)
Published in Journal of inherited metabolic disease (01.03.2024)
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Journal Article
Glycolate oxidase inhibition by lumasiran varies between patients with primary hyperoxaluria type 1
Garrelfs, Sander F., Metry, Elisabeth L., van Harskamp, Dewi, Vaz, Frederic M., van den Akker, Chris H.P., Schierbeek, Henk, Groothoff, Jaap W., Oosterveld, Michiel J.S.
Published in Kidney international (01.05.2023)
Published in Kidney international (01.05.2023)
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Journal Article
Successful kidney-alone transplantation in a patient with PH1 on combination RNA-interference therapy
Metry, Elisabeth L., Deesker, Lisa J., Garrelfs, Sander F., Oosterveld, Michiel J.S., Beekman, Kerensa M., Cornelissen, Marlies E.A., Koster-Kamphuis, Linda, Groothoff, Jaap W.
Published in Kidney international (01.07.2023)
Published in Kidney international (01.07.2023)
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Journal Article
Plasma oxalate and glycolate concentrations in dialysis patients with and without primary hyperoxaluria type 1
Metry, Elisabeth L, Garrelfs, Sander F, Peters-Sengers, Hessel, Vaz, Frederic M, Bijlsma, Joost A, Neradova, Aegida, Oosterveld, Michiel J S, Groothoff, Jaap W
Published in Nephrology, dialysis, transplantation (30.06.2023)
Published in Nephrology, dialysis, transplantation (30.06.2023)
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Journal Article
Corrigendum to “Successful kidney-alone transplantation in a patient with PH1 on combination RNA-interference therapy.” Kidney International, 2023;104:203–204
Metry, Elisabeth L., Deesker, Lisa J., Garrelfs, Sander F., Oosterveld, Michiel J.S., Beekman, Kerensa M., Cornelissen, Elisabeth A.M., Koster-Kamphuis, Linda, Groothoff, Jaap W.
Published in Kidney international (01.11.2023)
Published in Kidney international (01.11.2023)
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Journal Article
Development and Validation of a New Gas Chromatography–Tandem Mass Spectrometry Method for the Measurement of Enrichment of Glyoxylate Metabolism Analytes in Hyperoxaluria Patients Using a Stable Isotope Procedure
van Harskamp, Dewi, Garrelfs, Sander F, Oosterveld, Michiel J. S, Groothoff, Jaap W, van Goudoever, Johannes B, Schierbeek, Henk
Published in Analytical chemistry (Washington) (21.01.2020)
Published in Analytical chemistry (Washington) (21.01.2020)
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Journal Article
Endogenous Oxalate Production in Primary Hyperoxaluria Type 1 Patients
Garrelfs, Sander F, van Harskamp, Dewi, Peters-Sengers, Hessel, van den Akker, Chris H P, Wanders, Ronald J A, Wijburg, Frits A, van Goudoever, Johannes B, Groothoff, Jaap W, Schierbeek, Henk, Oosterveld, Michiel J S
Published in Journal of the American Society of Nephrology (01.12.2021)
Published in Journal of the American Society of Nephrology (01.12.2021)
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Journal Article
Transplantation outcomes in patients with primary hyperoxaluria: a systematic review
Metry, Elisabeth L., van Dijk, Liza M. M., Peters-Sengers, Hessel, Oosterveld, Michiel J.S., Groothoff, Jaap W., Ploeg, Rutger J., Stel, Vianda S., Garrelfs, Sander F.
Published in Pediatric nephrology (Berlin, West) (01.08.2021)
Published in Pediatric nephrology (Berlin, West) (01.08.2021)
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Journal Article
The Ocular Phenotype in Primary Hyperoxaluria Type 1
Birtel, Johannes, Herrmann, Philipp, Garrelfs, Sander F., Dulz, Simon, Atiskova, Yevgeniya, Diederen, Roselie M., Gliem, Martin, Brinkert, Florian, Holz, Frank G., Boon, Camiel J.F., Hoppe, Bernd, Charbel Issa, Peter
Published in American journal of ophthalmology (01.10.2019)
Published in American journal of ophthalmology (01.10.2019)
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Journal Article
Long-Term Transplantation Outcomes in Patients With Primary Hyperoxaluria Type 1 Included in the European Hyperoxaluria Consortium (OxalEurope) Registry
Metry, Elisabeth L., Garrelfs, Sander F., Peters-Sengers, Hessel, Hulton, Sally-Anne, Acquaviva, Cecile, Bacchetta, Justine, Beck, Bodo B., Collard, Laure, Deschênes, Georges, Franssen, Casper, Kemper, Markus J., Lipkin, Graham W., Mandrile, Giorgia, Mohebbi, Nilufar, Moochhala, Shabbir H., Oosterveld, Michiel J.S., Prikhodina, Larisa, Hoppe, Bernd, Cochat, Pierre, Groothoff, Jaap W.
Published in Kidney international reports (01.02.2022)
Published in Kidney international reports (01.02.2022)
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Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1
Hulton, Sally A., Groothoff, Jaap W., Frishberg, Yaacov, Koren, Michael J., Overcash, J. Scott, Sellier-Leclerc, Anne-Laure, Shasha-Lavsky, Hadas, Saland, Jeffrey M., Hayes, Wesley, Magen, Daniella, Moochhala, Shabbir H., Coenen, Martin, Simkova, Eva, Garrelfs, Sander F., Sas, David J., Meliambro, Kristin A., Ngo, Taylor, Sweetser, Marianne T., Habtemariam, Bahru A., Gansner, John M., McGregor, Tracy L., Lieske, John C.
Published in Kidney international reports (01.03.2022)
Published in Kidney international reports (01.03.2022)
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Journal Article
Primary hyperoxaluria in populations of Pakistan origin: results from a literature review and two major registries
Talati, Jamsheer Jehangir, Hulton, Sally-Anne, Garrelfs, Sander F., Aziz, Wajahat, Rao, Shoaib, Memon, Amanullah, Nazir, Zafar, Biyabani, Raziuddin, Qazi, Saqib, Azam, Iqbal, Khan, Aysha Habib, Ahmed, Jamil, Jafri, Lena, Zeeshan, Mohammad
Published in Urolithiasis (01.04.2018)
Published in Urolithiasis (01.04.2018)
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Journal Article
Determinants of Kidney Failure in Primary Hyperoxaluria Type 1: Findings of the European Hyperoxaluria Consortium
Metry, Elisabeth L., Garrelfs, Sander F., Deesker, Lisa J., Acquaviva, Cecile, D’Ambrosio, Viola, Bacchetta, Justine, Beck, Bodo B., Cochat, Pierre, Collard, Laure, Hogan, Julien, Ferraro, Pietro Manuel, Franssen, Casper F.M., Harambat, Jérôme, Hulton, Sally-Anne, Lipkin, Graham W., Mandrile, Giorgia, Martin-Higueras, Cristina, Mohebbi, Nilufar, Moochhala, Shabbir H., Neuhaus, Thomas J., Prikhodina, Larisa, Salido, Eduardo, Topaloglu, Rezan, Oosterveld, Michiel J.S., Groothoff, Jaap W., Peters-Sengers, Hessel
Published in Kidney international reports (01.10.2023)
Published in Kidney international reports (01.10.2023)
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Web Resource
Improved Outcome of Infantile Oxalosis Over Time in Europe: Data From the OxalEurope Registry
Deesker, Lisa J., Garrelfs, Sander F., Mandrile, Giorgia, Oosterveld, Michiel J.S., Cochat, Pierre, Deschênes, Georges, Harambat, Jérôme, Hulton, Sally-Anne, Gupta, Asheeta, Hoppe, Bernd, Beck, Bodo B., Collard, Laure, Topaloglu, Rezan, Prikhodina, Larisa, Salido, Eduardo, Neuhaus, Thomas, Groothoff, Jaap W., Bacchetta, Justine
Published in Kidney international reports (01.07.2022)
Published in Kidney international reports (01.07.2022)
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