Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial

Authors: Saland, Jeffrey M., Lieske, John C., Groothoff, Jaap W., Frishberg, Yaacov, Shasha-Lavsky, Hadas, Magen, Daniella, Moochhala, Shabbir H., Simkova, Eva, Coenen, Martin, Hayes, Wesley, Hogan, Julien, Sellier-Leclerc, Anne-Laure, Willey, Richard, Gansner, John M., Hulton, Sally-Anne

Source: In Kidney International Reports July 2024 9(7):2037-2046

Associations Between Prematurity, Birthweight, and Adolescence Blood Pressure in a Nationwide Cohort

Authors: Tzvi-Behr, Shimrit, Greenstein, Lucy B., Ben-Shalom, Efrat, Frishberg, Yaacov, Cohen, Sharon O.

Source: In Kidney International Reports May 2024 9(5):1228-1235

Dialysis in Israeli Children between 1990 and 2020: Trends and International Comparisons

Authors: Regev-Epstein, Lilach C., Frishberg, Yaacov, Davidovits, Miriam, Landau, Daniel, Magen, Daniella, Weismann, Irit, Stern-Zimmer, Michal, Beckerman, Pazit, Keinan-Boker, Lital, Calderon-Margalit, Ronit

Source: Clinical journal of the American Society of Nephrology. 18(3):363-373

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN632599077&indx=1&recIds=ETOCRN632599077

Diagnostic Utility of Exome Sequencing Among Israeli Children With Kidney Failure

Authors: Ben-Moshe, Yishay, Shlomovitz, Omer, Atias-Varon, Danit, Haskin, Orly, Ben-Shalom, Efrat, Shasha Lavsky, Hadas, Volovelsky, Oded, Mane, Shrikant, Ben-Ruby, Dror, Chowers, Guy, Skorecki, Karl, Borovitz, Yael, Kagan, Maayan, Mor, Nofar, Khavkin, Yulia, Tzvi-Behr, Shimrit, Pollack, Shirley, Toder, Moran Plonsky, Geylis, Michael, Schnapp, Aviad, Becker-Cohen, Rachel, Weissman, Irith, Schreiber, Ruth, Davidovits, Miriam, Frishberg, Yaacov, Magen, Daniella, Barel, Ortal, Vivante, Asaf

Source: In Kidney International Reports October 2023 8(10):2126-2135

Acute glomerulonephritis with concurrent suspected bacterial pneumonia – is it the tip of the iceberg?

Authors: Tzvi-Behr, Shimrit¹ shimrittb@szmc.org.il, Frishberg, Yaacov¹, Megged, Orli¹, Weinbrand-Goichberg, Jenny¹, Becher-Cohen, Rachel¹, Terespolsky, Hadass¹, Rinat, Choni¹, Choshen, Sapir¹, Ben-Shalom, Efrat¹

Source: Pediatric Nephrology. Apr2024, Vol. 39 Issue 4, p1143-1147. 5p.

Subject Terms: *PNEUMONIA, *RISK assessment, *DESCRIPTIVE statistics, *GLOMERULONEPHRITIS, *DISEASE risk factors, *DISEASE complications, *CHILDREN

The genetic landscape and clinical spectrum of nephronophthisis and related ciliopathies

Authors: Attié-Bitach, Tania, Comier-Daire, Valerie, Rozet, Jean-Michel, Frishberg, Yaacov, Llanas, Brigitte, Broyer, Michel, Mohsin, Nabil, Macher, Marie-Alice, Philip, Nicole, Baudouin, Véronique, Brackman, Damian, Loirat, Chantal, Charbit, Marina, Dehennault, Maud, Guyot, Claude, Bataille, Pierre, Elting, Mariet, Deschenes, Georges, Gropman, Andrea, Guest, Geneviève, Gagnadoux, Marie-France, Nicoud, Philippe, Cochat, Pierre, Ranchin, Bruno, Bensman, Albert, Guerrot, Anne-Marie, Knebelmann, Bertrand, Bilge, Ilmay, Bruno, Danièle, Burtey, Stéphane, Rouvière, Caroline Rousset, Caudwell, Valérie, Morin, Denis, Dollfus, Hélène, Maisin, Anne, Hamel, Christian, Bieth, Eric, Gie, Sophie, Goodship, Judith, Roussey, Gwenaelle, La Selve, Hermine, Nivet, Hubert, Bessenay, Lucie, Caillez, Mathilde, Palcoux, Jean Bernard, Benoît, Stéphane, Dubot, Philippe, Fila, Marc, Giuliano, Fabienne, Iftene, Daouya, Kessler, Michele, Kwon, Theresa, Lahoche, Anine, Laurent, Audrey, Leclerc, Anne-Laure, Milford, David, Neuhaus, Thomas, Odent, Sylvie, Eckart, Philippe, Chauveau, Dominique, Niaudet, Patrick, Repetto, Horacio, Taque, Sophie, Bruel, Alexandra, Noel-Botte, Alexandra, Launay, Emma Allain, Allard, Lisa, Anlicheau, Dany, Adra, Anne-Laure, Garnier, Arnaud, Nagra, Arvind, Baatard, Remy, Bacchetta, Justine, Sadikoglu, Banu, Barnerias, Christine, Barthelemy, Anne, Basel, Lina, Bassilios, Nader, Ben Maiz, Hedi, Ben Moussa, Fatma, Benmati, Faïza, Berthaud, Romain, Bertholet, Aurélia, Blanchier, Dominique, Boffa, Jean Jacques, Bouchireb, Karim, Bouhabel, Ihab, Boukerroucha, Zakaria, Bourdat-Michel, Guylhène, Boute, Odile, Brochard, Karine, Caumes, Roseline, Elalaoui, Siham Chafai, Chamontin, Bernard, Chastang, Marie Caroline, Pietrement, Christine, Richer, Christine, Legendre, Christophe, Dahan, Karin, Dalla-Vale, Fabienne, Thibaudin, Damien, Dauvergne, Maxime, Davourie, Salandre, Debeukelaer, Martin, Delbet, Jean Daniel, Deltas, Constantinos, Graber, Denis, Devillars, Nadège, Diouf, Boucar, Fenzy, Martine Doco, André, Jean-Luc, Joly, Dominique, Fryer, Alan, Albano, Laetitia, Cassuto, Elisabeth, Pincon, Aline, Medeira, Ana, Chaussenot, Annabelle, Mensire-Marinier, Anne, Bouissou, Francois, Decramer, Stephane, Bottani, Armand, Hummel, Aurélie, Karras, Alexandre, Katz, Avi, Azema, Christine, Janbon, Bénédicte, Roussel, Bernard, Bonniol, Claude, Mariat, Christiophe, Champion, Gérard, Chantreuil, Deborah, Chassaing, Nicolas, Mousson, Christiane, Baudeau, Christine, Cuntz, Delphine Hafdar, Mignot, Cyril, Dehoux, Laurene, Lacombe, Didier, Hannedouche, Thierry, Mérieau, Elodie, Charlin, Emmanuelle, Gauthier, Eric, Plasse, Florent, Faguer, Stanislas, Lebas, Fanny, Demurger, Florence, Emma, Francesco, Cartault, François, Dumont, Geneviève, Godefroid, Nathalie, Guigonis, Vincent, Hillaire, Sophie, Groothoff, Jaap, Dudley, Jan, Jourde-Chiche, Noémie, El Karoui, Khalil, Krid, Saoussen, Coudert, Krier, Bencheick, Larbi, Yver, Laurent, Lavocat, Marie-Pierre, De Sagazan, Le Monies, Leroy, Valerie, Thibaudin, Lise, Ingulli, Liz, Gwanmesia, Lorraine, Burglen, Lydie, Saïd-Menthon, Marie-Hélène, Carrera, Marta, Nizon, Mathilde, Melander, Catherine, Foulard, Michel, Blayo, Monique, Prinseau, Jacques, Jay, Nadine, Brun, Nathalie, Camille, Nicolas, Nobili, François, Devuyst, Olivier, Ben Brahim, Ouafa, Parvex, Paloma, Sabourin, Laurence Perrin, Blanc, Philippe, Vanhille, Philippe, Galichon, Pierre, Pierrepont, Sophie, Planquois, Vincent, Poussard, Gwenaelle, Noble, Claire Pouteil, Allal, Radia, Bernard, Raphaelle, Mounet, Raynaud, Cahen, Rémi, Touraine, Renaud, Rigothier, Claire, Ryckewaert, Amélie, Sacquepee, Mathieu, El Chehadeh, Salima, Samaille, Charlotte, Haq, Shuman, Simckes, Ari, Lanoiselée, Stéphanie, Tellier, Stephanie, Subra, Jean-François, Cloarec, Sylvie, Tenenbam, Julie, Lamy, Thomas, Garraud, Valérie Drouin, Valette, Huguette, Meyssonnier, Vanina, Vargas-Poussou, Rosa, Snajer, Yves, Durault, Sandrine, Plaisier, Emmanuelle, Berard, Etienne, Fakhouri, Fadi, Louillet, Ferielle, Finielz, Paul, Fischbach, Michel, Foliguet, Bernard, Francois-Pradier, Hélène, Garaix, Florentine, Gerard, Marion, Rizzoni, Gianfranco, Gilbert, Brigitte, Glotz, Denis, Dubrasquet, Astrid Godron, Grünfeld, Jean-Pierre, Bollee, Guillaume, Hall, Michelle, Hansson, Sverker, Haye, Damien, Taffin, Hélène, Hildebrandt, Friedhelm, Hourmand, Maryvonne, Kayserili, Hümya, Tack, Ivan, Jacquemont, Marie Line, Fabre-Teste, Jennifer, Kashtan, Cliff, Van Hoeck, Kkoen, Klein, Alexandre, Knefati, Yannick, Knoers, Nine, Konrad, Martin, Lachaux, Alain, Landru, Isabelle, Landthaler, Gilbert, Lang, Philippe, Le Pogamp, Patrick, Legris, Tristan, Didailler, Catherine, Lobbedez, Thierry, de Parscau, Loïc, Pinson, Lucile, Maheut, Hervé, Duval-Arnould, Marc, Rio, Marlène, Gubler, Marie-Claire, Merville, Pierre, Mestrallet, Guillaume, Meunier, Maite, Moreau, Karine, Harambat, Jérôme, Morgan, Graeme, Mourad, Georges, Stuber, Niksic, Boespflug-Tanguy, Odile, Dunand, Olivier, Niel, Olivier, Ouali, Nacera, Malvezzi, Paolo, Jaoude, Pauline Abou, Pelletier, Solenne, Peltier, Julie, Petersen, M.B., Michel, Philippe, Rémy, Philippe, Philit, Jean-Baptiste, Pichault, Valérie, Billette de Villemeur, Thierry, Boudailliez, Bernard, Leheup, Bruno, Dossier, Claire, Djeddi, Djamal-Dine, Berland, Yves, Hurault de Ligny, Bruno, Rigden, Susan, Robino, Christophe, Rossi, Annick, Sarnacki, Sabine, Saidani, Messaoud, Sartorius, Albane Brodin, Schäfer, Elise, Laszlo, Sztriha, Thouret, Marie-Christine, Thuillier-Lecouf, Angélique, Trachtman, Howard, Trivin, Claire, Tsimaratos, Michel, Van Damme-Lombaerts, Rita, Willems, Marjolaine, Youssef, Michel, Zaloszyc, Ariane, Zawodnik, Alexis, Ziliotis, Marie-Julia, Petzold, Friederike, Billot, Katy, Chen, Xiaoyi, Henry, Charline, Filhol, Emilie, Martin, Yoann, Avramescu, Marina, Douillet, Maxime, Morinière, Vincent, Krug, Pauline, Jeanpierre, Cécile, Tory, Kalman, Boyer, Olivia, Burgun, Anita, Servais, Aude, Salomon, Remi, Benmerah, Alexandre, Heidet, Laurence, Garcelon, Nicolas, Antignac, Corinne, Zaidan, Mohamad, Saunier, Sophie

Source: In Kidney International August 2023 104(2):378-387

Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial

Authors: Michael, Mini, Groothoff, Jaap W., Shasha-Lavsky, Hadas, Lieske, John C., Frishberg, Yaacov, Simkova, Eva, Sellier-Leclerc, Anne-Laure, Devresse, Arnaud, Guebre-Egziabher, Fitsum, Bakkaloglu, Sevcan A., Mourani, Chebl, Saqan, Rola, Singer, Richard, Willey, Richard, Habtemariam, Bahru, Gansner, John M., Bhan, Ishir, McGregor, Tracy, Magen, Daniella

Source: In American Journal of Kidney Diseases February 2023 81(2):145-155

Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1

Authors: 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.

Source: Kidney international reports. 7(3):494-506

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100157613610.0x000001&indx=1&recIds=ETOCvdc_100157613610.0x000001

Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children

Authors: Bacchetta, Justine, Baudouin, Véronique, Becker-Cohen, Rachel, Tzvi Behr, Shimrit, Ben-Shalom, Efrat, Berdaguer, Maria, Bockenhauer, Detlef, Cochat, Pierre, Coenen, Martin, Cramer, Carl H., Deschênes, Georges, Dossier, Claire, Doye, Emilie, Feldman, Liat Feraru, Hohenadel, Maximilian, Kaguelidou, Florentia, Zebegret, Irina Libinson, Lieske, John C., Maisin, Anne, Milliner, Dawn S., Plonsky Toder, Moran, Pollack, Shirley, Portefaix, Aurélie, Ranchin, Bruno, Rinat, Choni, Safdar, Adnan, Schalk, Gesa, Srivaths, Poyyapakkam R., Tran, Cheryl L., Van't Hoff, William, Weinbrand-Goichberg, Jenny, Weissman, Irith, Sas, David J., Magen, Daniella, Hayes, Wesley, Shasha-Lavsky, Hadas, Michael, Mini, Schulte, Indra, Sellier-Leclerc, Anne-Laure, Lu, Jiandong, Seddighzadeh, Ali, Habtemariam, Bahru, McGregor, Tracy L., Fujita, Kenji P., Frishberg, Yaacov

Source: In Genetics in Medicine March 2022 24(3):654-662

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1

Authors: Frishberg, Yaacov, Deschênes, Georges, Groothoff, Jaap W., Hulton, Sally-Anne, Magen, Daniella, Harambat, Jérôme, van't Hoff, William G., Lorch, Ulrike, Milliner, Dawn S., Lieske, John C., Haslett, Patrick, Garg, Pushkal P., Vaishnaw, Akshay K., Talamudupula, Sandeep, Lu, Jiandong, Habtemariam, Bahru A., Erbe, David V., McGregor, Tracy L., Cochat, Pierre

Source: Clinical journal of the American Society of Nephrology. 16(7):1025-1036

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100192340012.0x000001&indx=1&recIds=ETOCvdc_100192340012.0x000001

Mutations in HOGA1 do Not Confer a Dominant Phenotype Manifesting as Kidney Stone Disease

Authors: Bar, Roi, Ben-Shalom, Efrat, Duvdevani, Mordechai, Belostotsky, Ruth, Pollak, Martin R, Mount, David B., Bar-Gal, Ruth, Gnessin, Ehud, Tzur, Shay, Curhan, Gary C, Frishberg, Yaacov

Source: Journal of urology. 205(5):1394-1399

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100184810504.0x000001&indx=1&recIds=ETOCvdc_100184810504.0x000001

Phase ½ Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial

Authors: Frishberg, Yaacov, Deschênes, Georges, Groothoff, Jaap W., Hulton, Sally-Anne, Magen, Daniella, Harambat, Jérôme, Hoff, William G. van't, Lorch, Ulrike, Milliner, Dawn S., Lieske, John C.

Source: Clinical journal of the American Society of Nephrology. 16(7):1025-1036

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN627065083&indx=1&recIds=ETOCRN627065083

Diagnosis and management of primary hyperoxalurias: best practices.

Authors: Michael, Mini, Harvey, Elizabeth, Milliner, Dawn S., Frishberg, Yaacov, Sas, David J., Calle, Juan, Copelovitch, Lawrence, Penniston, Kristina L., Saland, Jeffrey, Somers, Michael J. G., Baum, Michelle A.

Source: Pediatric Nephrology; Nov2024, Vol. 39 Issue 11, p3143-3155, 13p

Subject Terms: KIDNEY failure, GENE therapy, KIDNEY transplantation, INBORN errors of carbohydrate metabolism, KIDNEY stones, URINARY calculi, HEMODIALYSIS, KIDNEY calcification, ALGORITHMS, LIVER transplantation, SYMPTOMS

Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial.

Authors: Frishberg, Yaacov, Hayes, Wesley, Shasha-Lavsky, Hadas, Sas, David J., Michael, Mini, Sellier-Leclerc, Anne-Laure, Hogan, Julien, Willey, Richard, Gansner, John M., Magen, Daniella

Source: Frontiers in Pediatrics; 2024, p01-11, 11p

Opportunities in Primary and Enteric Hyperoxaluria at the Cross-Roads Between the Clinic and Laboratory

Authors: Cellini, Barbara, Baum, Michelle A., Frishberg, Yaacov, Groothoff, Jaap W., Harris, Peter C., Hulton, Sally A., Knauf, Felix, Knight, John, Lieske, John C., Lowther, W. Todd, Moochhala, Shabbir, Nazzal, Lama, Tasian, Gregory E., Whittamore, Jonathan M., Sas, David J.

Source: In Kidney International Reports November 2024 9(11):3083-3096

FC070: Lumasiran for Patients with Primary Hyperoxaluria Type 1 with Impaired Kidney Function: Data from the 6-Month Analysis of the Phase 3 Illuminate-C Trial

Authors: Groothoff, Jaap, Michael, Mini, Shasha-Lavsky, Hadas, Lieske, John, Frishberg, Yaacov, Simkova, Eva, Sellier-Leclerc, Anne-Laure, Arnaud, Devresse, Guebre-Egziabher, Fitsum, Azime Bakkaloglu, Sevcan, Mourani, Chebl, Saqan, Rola, Singer, Richard, Willey, Richard, Habtemariam, Bahru, Bhan, Ishir, Gansner, John, Magen, Daniella

Source: Nephrology dialysis transplantation. 37(Supplement 3)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100152559171.0x000001&indx=1&recIds=ETOCvdc_100152559171.0x000001

Translation inhibition corrects aberrant localization of mutant alanine-glyoxylate aminotransferase: possible therapeutic approach for hyperoxaluria

Authors: Belostotsky, Ruth, Lyakhovetsky, Roman, Sherman, Michael Y., Shkedy, Fanny, Tzvi-Behr, Shimrit, Bar, Roi, Hoppe, Bernd, Reusch, Björn, Beck, Bodo B., Frishberg, Yaacov

Source: Journal of molecular medicine. 96(7):621-630

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100118090828.0x000001&indx=1&recIds=ETOCvdc_100118090828.0x000001

Metabolite diagnosis of primary hyperoxaluria type 3

Authors: Greed, Lawrence, Willis, Frank, Johnstone, Lilian, Teo, Sharon, Belostotsky, Ruth, Frishberg, Yaacov, Pitt, James

Source: Pediatric nephrology. 33(8):1443-1446

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100125314163.0x000001&indx=1&recIds=ETOCvdc_100125314163.0x000001

Left ventricular mass and cardiac function in pediatric dialysis patients

Authors: Rinat, Choni, Nir, Amiram, Frishberg, Yaacov

Source: In Progress in Pediatric Cardiology June 2016 41:75-82

Novel therapeutic approaches for the primary hyperoxalurias.

Authors: Belostotsky, Ruth¹, Frishberg, Yaacov^1,2 yaacovf@ekmd.huji.ac.il

Source: Pediatric Nephrology. Sep2021, Vol. 36 Issue 9, p2593-2606. 14p. 2 Diagrams, 1 Chart.

Subject Terms: *CHRONIC kidney failure, *GENETIC mutation, *METABOLISM, *DRUG design, *KIDNEY transplantation, *INBORN errors of carbohydrate metabolism, *OXALIC acid, *MOLECULAR structure, *LIVER transplantation, *GENETIC counseling, *BIOTECHNOLOGY, *SYMPTOMS