Long-term safety and efficacy of pegunigalsidase alfa: A multicenter extension study in adult patients with Fabry disease

被引：0

作者：

Hughes, Derralynn ^{[1
]}

Gonzalez, Derlis ^{[2
]}

Maegawa, Gustavo ^{[3
]}

Bernat, John ^{[4
]}

Holida, Myrl ^{[4
]}

Giraldo, Pilar ^{[5
]}

Atta, Mohamed ^{[6
]}

Chertkoff, Raul ^{[7
]}

Alon, Sari ^{[7
]}

Almon, Einat B. ^{[7
]}

Rocco, Rossana ^{[8
]}

Goker-Alpan, Ozlem ^{[9
]}

机构：

[1] UCL, London, England

[2] Inst Privado Hematol & Invest Clin, Asuncion, Paraguay

[3] Univ Florida, Gainesville, FL USA

[4] Univ Iowa Hlth Care, Iowa City, IA USA

[5] Hosp Dia Quiron, Zaragoza, Spain

[6] Johns Hopkins Sch Med, Baltimore, MD USA

[7] Protalix Biotherapeut, Carmiel, Israel

[8] Chiesi Farmaceut SpA, Parma, Italy

[9] Lysosomal Disorders Res & Treatment Unit, Fairfax, VA USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2021.11.143

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

128

引用

页码：S58 / S58

页数：1

共 50 条

[21] Safety and efficacy of pegunigalsidase alfa, every 4 weeks, in Fabry disease: Results from the phase 3, open-label, BRIGHT study
Bernat, John
Holida, Myrl
Longo, Nicola
Goker-Alpan, Ozlem
Wallace, Eric
Deegan, Patrick
Nedd, Khan
Tondel, Camilla
Eyskens, Francois
Feldt-Rasmussen, Ulla
Pisani, Antonio
Rocco, Rossana
Almon, Einat
Alon, Sari
Chertkoff, Raul
Warnock, David
Waldek, Stephen
Wilcox, William
GENETICS IN MEDICINE, 2022, 24 (03) : S91 - S92
[22] Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease
Zimran, Ari
Duran, Gloria
Mehta, Atul
Giraldo, Pilar
Rosenbaum, Hanna
Giona, Fiorina
Amato, Dominick J.
Petakov, Milan
Munoz, Eduardo Terreros
Solorio-Meza, Sergio Eduardo
Cooper, Peter A.
Varughese, Sheeba
Chertkoff, Raul
Brill-Almon, Einat
AMERICAN JOURNAL OF HEMATOLOGY, 2016, 91 (07) : 656 - 660
[23] Long-term efficacy and safety results of taliglucerase alfa through 5 years in adult treatment-naive patients with Gaucher disease
Zimran, Ari
Duran, Gloria
Giraldo, Pilar
Rosenbaum, Hanna
Giona, Fiorina
Petakov, Milan
Terreros Munoz, Eduardo
Eduardo Solorio-Meza, Sergio
Cooper, Peter A.
Varughese, Sheeba
Alon, Sari
Chertkoff, Raul
BLOOD CELLS MOLECULES AND DISEASES, 2019, 78 : 14 - 21
[24] Long-term efficacy and safety results of taliglucerase alfa through 5 years in adult treatment-naive patients with Gaucher disease
Chertkoff, Raul
Zimran, Ari
Duran, Gloria
Giraldo, Pilar
Rosenbaum, Hanna
Giona, Fiorina
Petakov, Milan
Eduardo Solorio-Meza, Sergio
Cooper, Peter A.
Varughese, Sheeba
Alon, Sari
Brill-Almon, Einat
MOLECULAR GENETICS AND METABOLISM, 2016, 117 (02) : S34 - S35
[25] 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
MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S159 - S160
[26] A long-term, multicenter study of the efficacy and safety of paricalcitol in end-stage renal disease
Lindberg, J
Martin, KJ
González, EA
Acchiardo, SR
Valdin, JR
Soltanek, C
CLINICAL NEPHROLOGY, 2001, 56 (04) : 315 - 323
[27] Long-Term Safety and Efficacy of Intracerebroventricular Enzyme Replacement Therapy with Cerliponase Alfa in Children with CLN2 Disease: 2 Year Results from an Ongoing Multicenter Extension Study
Jacoby, David
Schulz, Angela
Specchio, Nicola
Reyes, Emily de Los
Gissen, Paul
Cahan, Heather
Ajayi, Temitayo
ANNALS OF NEUROLOGY, 2017, 82 : S165 - S165
[28] Long-term Safety and Efficacy of Intracerebroventricular Enzyme Replacement Therapy with Cerliponase Alfa in Children with CLN2 Disease: Two Year Results from an Ongoing Multicenter Extension Study
de Los Reyes, E.
Schulz, A.
Specchio, N.
Gissen, P.
Lehwald, L.
Cahan, H.
Slasor, P.
Ajayi, T.
Jacoby, D.
ANNALS OF NEUROLOGY, 2018, 84 : S351 - S351
[29] Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study
Schulz, Angela
Specchio, Nicola
Gissen, Paul
de los Reyes, Emily
Cahan, Heather
Slasor, Peter
Ajayi, Temitayo
Jacoby, David
MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S120 - S120
[30] Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study
Schulz, Angela
Specchio, Nicola
Gissen, Paul
de los Reyesd, Emily
Cahan, Heather
Slasor, Peter
Ajayi, Temitayo
Jacoby, David
MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S126 - S127

← 1 2 3 4 5 →