Treatable lysosomal storage diseases in the advent of disease‐specific therapy von Peters, Heidi, Ellaway, Carolyn, Nicholls, Kathleen, Reardon, Katrina, Szer, Jeff

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Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry von Beck, Michael, Ramaswami, Uma, Hernberg-Ståhl, Elizabeth, Hughes, Derralynn A, Kampmann, Christoph, Mehta, Atul B, Nicholls, Kathleen, Niu, Dau-Ming, Pintos-Morell, Guillem, Reisin, Ricardo, West, Michael L, Schenk, Jörn, Anagnostopoulou, Christina, Botha, Jaco, Giugliani, Roberto

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The Effect of Fabry Disease Therapy on Bone Mineral Density von Aitken, Tess, Tiong, Mark K, Talbot, Andrew S, Ruderman, Irene, Nicholls, Kathleen M

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Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease? von Hughes, Derralynn A, Aguiar, Patrício, Lidove, Olivier, Nicholls, Kathleen, Nowak, Albina, Thomas, Mark, Torra, Roser, Vujkovac, Bojan, West, Michael L, Feriozzi, Sandro

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Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified D... von Hughes, Derralynn A, Aguiar, Patricio, Deegan, Patrick B, Ezgu, Fatih, Frustaci, Andrea, Lidove, Olivier, Linhart, Aleš, Lubanda, Jean-Claude, Moon, James C, Nicholls, Kathleen, Niu, Dau-Ming, Nowak, Albina, Ramaswami, Uma, Reisin, Ricardo, Rozenfeld, Paula, Schiffmann, Raphael, Svarstad, Einar, Thomas, Mark, Torra, Roser, Vujkovac, Bojan, Warnock, David G, West, Michael L, Johnson, Jack, Rolfe, Mark J, Feriozzi, Sandro

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Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial von Schiffmann, Raphael, Bichet, Daniel G, Jovanovic, Ana, Hughes, Derralynn A, Giugliani, Roberto, Feldt-Rasmussen, Ulla, Shankar, Suma P, Barisoni, Laura, Colvin, Robert B, Jennette, J Charles, Holdbrook, Fred, Mulberg, Andrew, Castelli, Jeffrey P, Skuban, Nina, Barth, Jay A, Nicholls, Kathleen

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Fabry in the older patient: Clinical consequences and possibilities for treatment von Lidove, Olivier, Barbey, Frédéric, Niu, Dau-Ming, Brand, Eva, Nicholls, Kathleen, Bizjajeva, Svetlana, Hughes, Derralynn A.

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Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified... von Hopkin, Robert J., Feldt-Rasmussen, Ulla, Germain, Dominique P., Jovanovic, Ana, Martins, Ana Maria, Nicholls, Kathleen, Ortiz, Alberto, Politei, Juan, Ponce, Elvira, Varas, Carmen, Weidemann, Frank, Yang, Meng, Wilcox, William R.

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Foot screening in a dialysis unit - a pilot educational project von Leo, Joan, Nicholls, Kathleen

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Sodium-glucose cotransporter 2 inhibitors reduce albuminuria in patients with Fabry disease: a real-world case series von Tsatsaronis, Anthea, Tiong, Mark, Nicholls, Kathleen, Ruderman, Irene

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Increased arterial stiffness is associated with high cardiovascular mortality in male Fabry patients von Nicholls, Kathleen

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The spectrum of podocyte injury in later onset (LO) variants of Fabry disease (FD) von Najafian, Behzad, Nicholls, Kathleen, West, Michael L., Tøndel, Camilla, Svarstad, Einar, Jovanovic, Ana, Mauer, Michael

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A 15-Year Perspective of the Fabry Outcome Survey von Giugliani, Roberto, Niu, Dau-Ming, Ramaswami, Uma, West, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Nicholls, Kathleen, Schenk, Jörn-Magnus, Beck, Michael

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Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat von Germain, Dominique P, Hughes, Derralynn A, Nicholls, Kathleen, Bichet, Daniel G, Giugliani, Roberto, Wilcox, William R, Feliciani, Claudio, Shankar, Suma P, Ezgu, Fatih, Amartino, Hernan, Bratkovic, Drago, Feldt-Rasmussen, Ulla, Nedd, Khan, Sharaf El Din, Usama, Lourenco, Charles M, Banikazemi, Maryam, Charrow, Joel, Dasouki, Majed, Finegold, David, Giraldo, Pilar, Goker-Alpan, Ozlem, Longo, Nicola, Scott, C. Ronald, Torra, Roser, Tuffaha, Ahmad, Jovanovic, Ana, Waldek, Stephen, Packman, Seymour, Ludington, Elizabeth, Viereck, Christopher, Kirk, John, Yu, Julie, Benjamin, Elfrida R, Johnson, Franklin, Lockhart, David J, Skuban, Nina, Castelli, Jeff, Barth, Jay, Barlow, Carrolee, Schiffmann, Raphael

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Genetics in chronic kidney disease: conclusions from a Kidney Disease: Improving Global Outcomes (KDIGO) Controversies Conference von Köttgen, Anna, Cornec-Le Gall, Emilie, Halbritter, Jan, Kiryluk, Krzysztof, Mallett, Andrew J., Parekh, Rulan S., Rasouly, Hila Milo, Sampson, Matthew G., Tin, Adrienne, Antignac, Corinne, Ars, Elisabet, Bergmann, Carsten, Bleyer, Anthony J., Bockenhauer, Detlef, Devuyst, Olivier, Florez, Jose C., Fowler, Kevin J., Franceschini, Nora, Fukagawa, Masafumi, Gale, Daniel P., Gbadegesin, Rasheed A., Goldstein, David B., Grams, Morgan E., Greka, Anna, Gross, Oliver, Guay-Woodford, Lisa M., Harris, Peter C., Hoefele, Julia, Hung, Adriana M., Knoers, Nine V.A.M., Kopp, Jeffrey B., Kretzler, Matthias, Lanktree, Matthew B., Lipska-Ziętkiewicz, Beata S., Nicholls, Kathleen, Nozu, Kandai, Ojo, Akinlolu, Parsa, Afshin, Pattaro, Cristian, Pei, York, Pollak, Martin R., Rhee, Eugene P., Sanna-Cherchi, Simone, Savige, Judy, Sayer, John A., Scolari, Francesco, Sedor, John R., Sim, Xueling, Somlo, Stefan, Susztak, Katalin, Tayo, Bamidele O., Torra, Roser, van Eerde, Albertien M., Weinstock, André, Winkler, Cheryl A., Wuttke, Matthias, Zhang, Hong, King, Jennifer M., Cheung, Michael, Jadoul, Michel, Winkelmayer, Wolfgang C., Gharavi, Ali G.

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Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study von Feldt-Rasmussen, Ulla, Hughes, Derralynn, Sunder-Plassmann, Gere, Shankar, Suma, Nedd, Khan, Olivotto, Iacopo, Ortiz, Damara, Ohashi, Toya, Hamazaki, Takashi, Skuban, Nina, Yu, Julie, Barth, Jay A., Nicholls, Kathleen

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Fabry‐specific treatment in Australia: time to align eligibility criteria with international best practices von Nicholls, Kathleen, Denaro, Charles, Tchan, Michel, Ellaway, Carolyn, Bratkovic, Drago, Campbell, Sheridan, Fookes, Megan, Thomas, Mark

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Clinical impact of genomic testing in patients with suspected monogenic kidney disease von Jayasinghe, Kushani, Stark, Zornitza, Kerr, Peter G., Gaff, Clara, Martyn, Melissa, Whitlam, John, Creighton, Belinda, Donaldson, Elizabeth, Hunter, Matthew, Jarmolowicz, Anna, Johnstone, Lilian, Krzesinski, Emma, Lunke, Sebastian, Lynch, Elly, Nicholls, Kathleen, Patel, Chirag, Prawer, Yael, Ryan, Jessica, See, Emily J., Talbot, Andrew, Trainer, Alison, Tytherleigh, Rigan, Valente, Giulia, Wallis, Mathew, Wardrop, Louise, West, Kirsty H., White, Susan M., Wilkins, Ella, Mallett, Andrew J., Quinlan, Catherine

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OP58 Challenges In Maintaining Up To Date Health Technology Assessments in Rare Diseases: Lessons From Fabry Disease In Australia von Nicholls, Kathleen, Denaro, Charles, Tchan, Michel, Ellaway, Carolyn, Bratkovic, Drago, Palmer, Hazel, Campbell, Sheridan, Fookes, Megan, Thomas, Mark

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AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Overview of clinical data from phase 1 and phase 2 studies von Thomas, Mark, Nicholls, Kathleen, Carnley, Benedict, Trame, Mirjam, Mason, Chris, Tavakkoli, Fatemeh, Keenan, Greg

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