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ILLUMINATE-A, une étude de phase 3 du lumasiran, un ARNi thérapeutique expérimental, chez les enfants et les adultes atteints d’hyperoxalurie primaire de type 1

Autor: Hadas Shasha-Lavsky, Kristin Meliambro, Jiandong Lu, Pierre Cochat, Gesa Schalk, John M. Gansner, Pushkal Garg, Georges Deschênes, Sander F. Garrelfs, John C. Lieske, Sally-Anne Hulton, Michael J. Koren, Yaacov Frishberg, Eva Simkova, William O'Riordan, Jaap W. Groothoff, Tracy L. McGregor, Jeffrey M. Saland, David J. Sas, Daniel Guido Fuster, Shabbir H. Moochhala, Daniella Magen, W van't Hoff

Publikováno v: Néphrologie & Thérapeutique. 16:262

Introduction L’hyperoxalurie primaire de type I (HP1) est une maladie genetique rare, caracterisee par une production hepatique excessive d’oxalate, une excretion urinaire elevee d’oxalate, des calculs renaux recurrents et une nephrocalcinose,

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::c75d909add7ace84632ad6cb01856e33
https://doi.org/10.1016/j.nephro.2020.07.036

The orthopaedic management of lower limb deformity in hypophosphataemic rickets

Autor: W van't Hoff, Deborah M. Eastwood, Detlef Bockenhauer, Jonathan Wright, A Horn

Publikováno v: Journal of Children's Orthopaedics

Background Many patients with X-linked hypophosphataemic rickets (X-LHPR) demonstrate significant lower limb deformity despite optimal medical management. This study evaluates the use of guided growth by means of hemi-epiphysiodesis to address corona

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bd161f6df1ddfaef6f01ae18fcaef82f
https://pubmed.ncbi.nlm.nih.gov/28904636

SUN-325 SAFETY AND EFFICACY OF LUMASIRAN, AN INVESTIGATIONAL RNA INTERFERENCE (RNAi) THERAPEUTIC, IN ADULT AND PEDIATRIC PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1

Autor: Bernd Hoppe, Pierre Cochat, Dawn S. Milliner, Jaap W. Groothoff, W van't Hoff, Georges Deschênes, Jérôme Harambat, Daniella Magen, John C. Lieske, Sally-Anne Hulton, Yaacov Frishberg

Publikováno v: Kidney International Reports. 4:S295

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::e0b7fb3edf3128ab29a1281663475931
https://doi.org/10.1016/j.ekir.2019.05.734

Paediatric nephrology II

Autor: K. Musial, D. Zwolinska, R. Pruthi, M. Sinha, A. Casula, M. Lewis, Y. Tse, H. Maxwell, C. O'Brien, C. Inward, E. Sharaf, F. Fadel, H. Bazaraa, R. Hegazy, R. Essam, B. Manickavasagar, R. Shroff, A. McArdle, S. Ledermann, V. Shaw, W. Van't Hoff, B. Paudyal, G. Prado, M. Schoeneman, M. K. Nepal, V. Feygina, V. Bansilal, H. Tawadrous, A. K. Mongia, A. Melk, D. Kracht, A. Doyon, R. Zeller, M. Litwin, A. Duzowa, B. Sozeri, A. Bayzit, S. Caliskan, U. Querfeld, E. Wuhl, F. Schaefer, B. Schmidt, N. Canpolat, M. Kara Acar, S. Pehlivan, M. Tasdemir, L. Sever, E. Nusken, C. Taylan, G. von Gersdorff, M. Schaller, C. Barth, J. Dotsch, P. Roomizadeh, A. Gheissari, A. Abedini, F. Garzotto, M. Zanella, J. Kim, R. Cena, M. Neri, F. Nalesso, A. Brendolan, C. Ronco, T. Celkan, S. Lacinel, A. Keser, A. Taner Elmas, Y. Tabel, S. Ipek, A. Karadag, O. Elmas, F. Ozyalin, A. Hoxha (Qosja), A. Gjyzari, E. Tushe, R. M. Said, M. A. Abdel Fattah, D. A. Soliman, S. Y. Mahmoud, M. Hattori, O. Uemura, H. Hataya, S. Ito, M. Hisano, T. Ohta, S. Fujinaga, T. Kise, Y. Goto, A. Matsunaga, T. Hashimoto, Y. Tsutsumi, N. Ito, T. Akizawa, S. Maher, B.-S. Cho, Y.-M. Choi, J.-S. Suh, F. Farid, I. El-Hakim, M. Salman, S. Rajnochova Bloudickova, O. Viklicky, T. Seeman, S. Yuksel, M. Caglar, T. Becerir, E. Tepeli, N. Calli Demirkan, N. Yalcin, A. Ergin, M. Hladik, R. Sigutova, F. Vsiansky, K. Safarcik, Z. Svagera, N. Abd El Monem Soliman, H. M. Bazaraa, M. M. Nabhan, A. M. Badr, M. Abd El Latif Shahin, P. Skrzypczyk, M. Panczyk-Tomaszewska, M. Roszkowska-Blaim, Z. Wawer, B. Bienias, M. Zajaczkowska, M. Szczepaniak, M. Pawlak-Bratkowska, M. Tkaczyk, K. Kilis-Pstrusinska, A. Jakubowska, L. Prikhodina, O. Ryzhkova, N. Poltavets, V. Polyakov

Publikováno v: Nephrology Dialysis Transplantation. 28:i517-i523

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::1c781814641b39dd66220c18bcaa5699
https://doi.org/10.1093/ndt/gft157