Results 21 to 30 of about 167,688 (127)

From diagnosis to disease-specific treatment: first experience with enzyme replacement therapy for Fabry disease in North Macedonia—a case series [PDF]

Frontiers in Medicine
BackgroundFabry disease is a rare X-linked lysosomal storage disorder caused by deficiency of α-galactosidase A, leading to progressive accumulation of globotriaosylceramide and Lyso-Gb3 across multiple organ systems.
Vlatko Karanfilovski, Igor G. Nikolov, Pavlina Dzekova Vidimliski, Svetlana Krstevska Balkanov, Galina Severova, Ana Stojanoska, Nikola Gjorgjievski   +6 more
doaj   +2 more sources

Current status of the immunogenicity of enzyme replacement therapy in fabry disease [PDF]

Orphanet Journal of Rare Diseases
In patients with Fabry disease (FD), treatment with enzyme replacement therapy (ERT), may trigger the formation of anti-drug antibodies (ADAs). The consequences of this immune reaction range from the transient appearance of clinically insignificant ADAs ...
Jorge F. Gómez-Cerezo, Julián Fernández-Martín, Miguel Ángel Barba-Romero, Rosario Sánchez-Martínez, Alvaro Hermida-Ameijeiras, Maria Camprodon-Gómez, Saida Ortolano, Mónica A. Lopez-Rodriguez   +7 more
doaj   +2 more sources

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

Drug Design, Development and Therapy, 2016
Ozlem Goker-Alpan,1 Nicola Longo,2 Marie McDonald,3 Suma P Shankar,4,5 Raphael Schiffmann,6 Peter Chang,7 Yinghua Shen,7 Arian Pano7 1Lysosomal Disorders Unit, Fairfax, VA, 2University of Utah, Salt Lake City, UT, 3Department of Pediatrics, Duke ...
Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A   +7 more
doaj   +1 more source

Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study [PDF]

Orphanet Journal of Rare Diseases
Background Enzyme replacement therapies (ERTs) approved for Fabry disease require infusions every 2 weeks (E2W). Pegunigalsidase alfa, a PEGylated ERT with a prolonged half-life vs. other ERTs, may allow extension of the dosing interval to every 4 weeks (
Myrl Holida, Aleš Linhart, Nicola Longo, Eric Wallace, Camilla Tøndel, Derralynn Hughes, David G. Warnock, Antonio Pisani, François Eyskens, Patrick Deegan, Ulla Feldt-Rasmussen, Ozlem Goker-Alpan, Ankit Mehta, Giovanni Piotti, Vito Fichera, Meng Wang, Raul Chertkoff, Stephen Waldek, William R. Wilcox, John A. Bernat   +19 more
doaj   +2 more sources

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. [PDF]

, 2023
BACKGROUND: Pegunigalsidase alfa is a PEGylated α-galactosidase A enzyme replacement therapy. BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with an annualised estimated ...
Kantola, Ilkka, Holida, Myrl, Knoll, Jasmine, Langeveld, Mirjam, Barshop, Bruce A, Kimonis, Virginia, Vujkovac, Bojan, Karaa, Amel, Tøndel, Camilla, Geberhiwot, Tarekegn, Jovanovic, Ana, Muriello, Michael, Deegan, Patrick B, Hughes, Derralynn A; https://orcid.org/, Waldek, Stephen, Ortiz, Damara, Giraldo, Pilar, Mehta, Ankit, Wallace, Eric L, Bernat, John, Pisani, Antonio, Nedd, Khan, Longo, Nicola, Goker-Alpan, Ozlem, Nowak, Albina; https://orcid.org/, et al, Wilcox, William R, Molnar, Maria Judit, Germain, Dominique Paul; https://orcid.org/, Linhart, Aleš; https://orcid.org/, Hopkin, Robert J   +30 more
core   +13 more sources

Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease. [PDF]

J Clin Med, 2022
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, which, without treatment, can cause significant renal dysfunction.
Cybulla M, Nicholls K, Feriozzi S, Linhart A, Torras J, Vujkovac B, Botha J, Anagnostopoulou C, West ML, FOS Study Group.   +9 more
europepmc   +3 more sources

Occurrence of Infusion Associated Reactions and Antidrug Antibodies in Enzyme Replacement Therapy for Fabry Disease and the Effect of Preventive Measures. [PDF]

J Inherit Metab Dis
Occurrence of infusion associated reactions and anti‐drug antibodies in enzyme replacement therapy for Fabry disease and the effect of preventive measures. ABSTRACT Male patients with the classical phenotype of Fabry disease (FD) are at risk of developing inhibiting antidrug antibodies (iADAs) against recombinant α‐galactosidase‐A, administered in the ...
Janssens M, van der Veen SJ, van Dussen L, van Kuilenburg ABP, Brands MMMG, Langeveld M.   +5 more
europepmc   +2 more sources

Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa

Molecular Therapy: Methods & Clinical Development, 2022
We analyzed the cross-reactivity of anti-drug antibodies (ADAs) against agalsidase-alfa and -beta from 49 patients with Fabry disease (FD) against the novel PEGylated enzyme pegunigalsidase-alfa (PRX-102).
Malte Lenders, Solvey Pollmann, Melina Terlinden, Eva Brand   +3 more
doaj   +1 more source

New insights in efficacy of different ERT dosages in Fabry disease: Switch and switch-back studies data following agalsidase beta shortage. Update of systematic review

Genetics in Medicine Open, 2023
In 2016, a systematic review and a meta-analysis of existing data on the effects of switch from agalsidase beta to alfa in patients with Fabry disease showed that the switch was well tolerated and associated with stable disease progression.
Eleonora Riccio, Carlo Garofalo, Ivana Capuano, Pasquale Buonanno, Guido Iaccarino, Teodolinda Di Risi, Massimo Imbriaco, Federica Riccio Cuomo, Antonio Pisani   +8 more
doaj   +1 more source

Assessment and impact of dose escalation on anti-drug antibodies in Fabry disease

Frontiers in Immunology, 2022
Enzyme replacement therapy (ERT) with recombinant α-galactosidase A (AGAL) can lead to the formation of neutralizing anti-drug antibodies (ADA), which significantly limit treatment efficacy in patients with Fabry disease (FD).
Malte Lenders, Eva Brand
doaj   +1 more source