Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1‐year Phase 1/2 clinical trial
Schiffmann, Raphael, Goker‐Alpan, Ozlem, Holida, Myrl, Giraldo, Pilar, Barisoni, Laura, Colvin, Robert B., Jennette, Charles J., Maegawa, Gustavo, Boyadjiev, Simeon A., Gonzalez, Derlis, Nicholls, Kathy, Tuffaha, Ahmad, Atta, Mohamed G., Rup, Bonita, Charney, Martha R., Paz, Alona, Szlaifer, Mali, Alon, Sari, Brill‐Almon, Einat, Chertkoff, Raul, Hughes, Derralynn
Published in Journal of inherited metabolic disease (01.05.2019)
Published in Journal of inherited metabolic disease (01.05.2019)
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Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study
Linhart, AleÅ, Dostálová, Gabriela, Nicholls, Kathy, West, Michael L, Tandel, Camilla, Jovanovic, Ana, Giraldo, Pilar, Vujkovac, Bojan, Geberhiwot, Tarekegn, Brill-Almon, Einat, Alon, Sari, Chertkoff, Raul, Rocco, Rossana, Hughes, Derralynn
Published in Orphanet journal of rare diseases (21.10.2023)
Published in Orphanet journal of rare diseases (21.10.2023)
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Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system
Shaaltiel, Yoseph, Bartfeld, Daniel, Hashmueli, Sharon, Baum, Gideon, Brill-Almon, Einat, Galili, Gad, Dym, Orly, Boldin-Adamsky, Swetlana A., Silman, Israel, Sussman, Joel L., Futerman, Anthony H., Aviezer, David
Published in Plant biotechnology journal (01.09.2007)
Published in Plant biotechnology journal (01.09.2007)
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A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation
Aviezer, David, Brill-Almon, Einat, Shaaltiel, Yoseph, Hashmueli, Sharon, Bartfeld, Daniel, Mizrachi, Sarah, Liberman, Yael, Freeman, Arnold, Zimran, Ari, Galun, Eithan
Published in PloS one (11.03.2009)
Published in PloS one (11.03.2009)
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Enzyme replacement therapy with taliglucerase alfa: 36‐month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase
Pastores, Gregory M., Shankar, Suma P., Petakov, Milan, Giraldo, Pilar, Rosenbaum, Hanna, Amato, Dominick J., Szer, Jeffrey, Chertkoff, Raul, Brill‐Almon, Einat, Zimran, Ari
Published in American journal of hematology (01.07.2016)
Published in American journal of hematology (01.07.2016)
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Journal Article
Long‐term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment‐naïve patients with Gaucher disease
Zimran, Ari, Durán, Gloria, Mehta, Atul, Giraldo, Pilar, Rosenbaum, Hanna, Giona, Fiorina, Amato, Dominick J., Petakov, Milan, Muñoz, Eduardo Terreros, Solorio‐Meza, Sergio Eduardo, Cooper, Peter A., Varughese, Sheeba, Chertkoff, Raul, Brill‐Almon, Einat
Published in American journal of hematology (01.07.2016)
Published in American journal of hematology (01.07.2016)
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Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease
Hughes, Derralynn, Gonzalez, Derlis, Maegawa, Gustavo, Bernat, John A, Holida, Myrl, Giraldo, Pilar, Atta, Mohamed G, Chertkoff, Raul, Alon, Sari, Almon, Einat Brill, Rocco, Rossana, Goker-Alpan, Ozlem
Published in Genetics in medicine (01.12.2023)
Published in Genetics in medicine (01.12.2023)
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Journal Article
Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease
Zimran, Ari, Brill-Almon, Einat, Chertkoff, Raul, Petakov, Milan, Blanco-Favela, Francisco, Muñoz, Eduardo Terreros, Solorio-Meza, Sergio E., Amato, Dominick, Duran, Gloria, Giona, Fiorina, Heitner, Rene, Rosenbaum, Hanna, Giraldo, Pilar, Mehta, Atul, Park, Glen, Phillips, Mici, Elstein, Deborah, Altarescu, Gheona, Szleifer, Mali, Hashmueli, Sharon, Aviezer, David
Published in Blood (24.11.2011)
Published in Blood (24.11.2011)
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Pegunigalsidase alfa, a novel PEGylated ERT, evaluated in Fabry disease patients with progressing kidney disease, RCT study design
Warnock, David G., Hughes, Derralynn, Alon, Sari, Chertkoff, Raul, Brill-Almon, Einat, Schiffmann, Raphael
Published in Molecular genetics and metabolism (01.02.2020)
Published in Molecular genetics and metabolism (01.02.2020)
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Assessment of immunogenicity from the pegunigalsidase alfa clinical trial program: Integrated analysis of de novo and treatment-boosted anti-drug antibodies
Bernat, John, Hughes, Derralynn, Linhart, Aleš, Hopkin, Robert J., Tøndel, Camilla, Warnock, David G., Rup, Bonita, Koulinska, Irene, Rocco, Rossana, Alon, Sari, Chertkoff, Raul, Almon, Einat Brill, Sakov, Anat, Wallace, Eric
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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Journal Article
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study
Wallace, Eric L, Goker-Alpan, Ozlem, Wilcox, William R, Holida, Myrl, Bernat, John, Longo, Nicola, Linhart, Aleš, Hughes, Derralynn A, Hopkin, Robert J, Tøndel, Camilla, Langeveld, Mirjam, Giraldo, Pilar, Pisani, Antonio, Germain, Dominique Paul, Mehta, Ankit, Deegan, Patrick B, Molnar, Maria Judit, Ortiz, Damara, Jovanovic, Ana, Muriello, Michael, Barshop, Bruce A, Kimonis, Virginia, Vujkovac, Bojan, Nowak, Albina, Geberhiwot, Tarekegn, Kantola, Ilkka, Knoll, Jasmine, Waldek, Stephen, Nedd, Khan, Karaa, Amel, Brill-Almon, Einat, Alon, Sari, Chertkoff, Raul, Rocco, Rossana, Sakov, Anat, Warnock, David G
Published in Journal of medical genetics (01.06.2024)
Published in Journal of medical genetics (01.06.2024)
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A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies
Holida, Myrl, Linhart, Aleš, Pisani, Antonio, Longo, Nicola, Eyskens, François, Goker-Alpan, Ozlem, Wallace, Eric, Deegan, Patrick, Tøndel, Camilla, Feldt-Rasmussen, Ulla, Hughes, Derralynn, Sakov, Anat, Rocco, Rossana, Almon, Einat Brill, Alon, Sari, Chertkoff, Raul, Warnock, David G, Waldek, Stephen, Wilcox, William R, Bernat, John A
Published in Journal of inherited metabolic disease (09.10.2024)
Published in Journal of inherited metabolic disease (09.10.2024)
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Journal Article
Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: Two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study
Bernat, John, Holida, Myrl D., Longo, Nicola, Goker-Alpan, Ozlem, Wallace, Eric, Deegan, Patrick B., Tøndel, Camilla, Eyskens, Francois J., Feldt-Rasmussen, Ulla, Hughes, Derralynn, Pisani, Antonio, Rocco, Rossana, Almon, Einat Brill, Alon, Sari, Chertkoff, Raul, Warnock, David G., Waldek, Stephen, Wilcox, William R.
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase
Pastores, Gregory M., Petakov, Milan, Giraldo, Pilar, Rosenbaum, Hanna, Szer, Jeffrey, Deegan, Patrick B., Amato, Dominick J., Mengel, Eugen, Tan, Ee Shien, Chertkoff, Raul, Brill-Almon, Einat, Zimran, Ari
Published in Blood cells, molecules, & diseases (01.12.2014)
Published in Blood cells, molecules, & diseases (01.12.2014)
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Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease
Zimran, Ari, Gonzalez-Rodriguez, Derlis Emilio, Abrahamov, Aya, Elstein, Deborah, Paz, Alona, Brill-Almon, Einat, Chertkoff, Raul
Published in Blood cells, molecules, & diseases (01.01.2015)
Published in Blood cells, molecules, & diseases (01.01.2015)
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Journal Article
Enhanced pharmacokinetics profile of pegunigalsidase alfa (PRX-102) supports once-monthly 2mg/kg dosing for the treatment of Fabry disease
Warnock, David G., Charney, Martha R., Hughes, Derralynn, Schiffmann, Raphael, Alon, Sari, Chertkoff, Raul, Brill-Almon, Einat
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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Journal Article
Switching from agalsidase alfa to pegunigalsidase alfa for treating Fabry disease: One year of treatment data from BRIDGE, a phase III open label study
Linhart, Ales, Dostalova, Gabriela, Nicholls, Kathy, West, Michael, Tøndel, Camilla, Jovanovic, Ana, Giraldo, Pilar, Vujkovac, Bojan, Hiwot, Tarekegn, Brill-Almon, Einat, Alon, Sari, Szlaifer, Mali, Chertkoff, Raul, Hughes, Derralynn
Published in Molecular genetics and metabolism (01.02.2020)
Published in Molecular genetics and metabolism (01.02.2020)
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Journal Article
Pegunigalsidase alfa, PEGylated α-galactosidase-A enzyme in development for the treatment of Fabry disease, shows correlation between renal GB3 inclusion clearance and reduction of plasma Lyso-GB3
Hughes, Derralynn, Boyd, Simeon, Giraldo, Pilar, Gonzales, Derlis, Holida, Myrl, Goker-Alpan, Ozlem, Atta, Mohamed G., Nicholls, Kathy, Schiffmann, Raphael, Tuffaha, Ahmad, Maegawa, Gustavo, Barisoni, Laura, Colvin, Robert, Jennette, J. Charles, Alon, Sari, Szlaifer, Mali, Brill-Almon, Einat, Chertkoff, Raul
Published in Molecular genetics and metabolism (01.02.2020)
Published in Molecular genetics and metabolism (01.02.2020)
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Journal Article
Pegunigalsidase alfa, a novel PEGylated ERT for Fabry disease: Two-year safety and efficacy follow up
Hughes, Derralynn, Giraldo, Pilar, Holida, Myrl, Goker-Alpan, Ozlem, Maegawa, Gustavo, Schiffmann, Raphael, Alon, Sari, Filipovich, Margarita, Szlaifer, Mali, Brill-Almon, Einat, Chertkoff, Raul
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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