Results 21 to 30 of about 744,838 (140)

Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice [PDF]

Clinical Pharmacology in Drug Development, 2021
Anibh M Das, Raphael Schiffmann, Richie Khanna   +2 more
exaly   +2 more sources

Evaluating the relationship between antidrug antibodies and efficacy and safety outcomes in patients with Fabry disease receiving enzyme replacement therapy: a systematic literature review [PDF]

Orphanet Journal of Rare Diseases
Background Fabry disease is a lysosomal storage disorder caused by deficient activity of the enzyme α-galactosidase A, resulting in progressive accumulation of globotriaosylceramide (Gb3) and widespread tissue and organ damage. Enzyme replacement therapy
Patricio Aguiar, John A. Bernat, Derralynn Hughes   +2 more
doaj   +2 more sources

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

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]

J Med Genet, 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 ...
Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG.   +35 more
europepmc   +11 more sources

Clinical outcomes in Fabry patients switching to agalsidase beta for renal ineffectiveness of the primary Fabry therapy: a single-centre analysis. [PDF]

Clin Kidney J
Background A treatment for classic Fabry disease (FD) should be defined effective on renal function when it maintains a decline of estimated glomerular filtration rate (eGFR)
Riccio E, Capuano I, Buonanno P, Iaccarino G, Cirami CL, Mignani R, Pieruzzi F, Pisani A.   +7 more
europepmc   +2 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

Rapid Immunochromatographic Detection of Serum Anti-α-Galactosidase A Antibodies in Fabry Patients after Enzyme Replacement Therapy. [PDF]

PLoS ONE, 2015
We developed an immunochromatography-based assay for detecting antibodies against recombinant α-galactosidase A proteins in serum. The evaluation of 29 serum samples from Fabry patients, who had received enzyme replacement therapy with agalsidase alpha ...
Sachie Nakano, Takahiro Tsukimura, Tadayasu Togawa, Toya Ohashi, Masahisa Kobayashi, Katsuyoshi Takayama, Yukuharu Kobayashi, Hiroshi Abiko, Masatsugu Satou, Tohru Nakahata, David G Warnock, Hitoshi Sakuraba, Futoshi Shibasaki   +12 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

Treatment switch from agalsidase beta to agalsidase alfa for Fabry disease

, 2023
Die lysosomale Speichererkrankung Morbus Fabry wird X-chromosomal rezessiv vererbt und führt durch eine Mutation des α-Galactosidase A-Gens zu einer fehlerhaften Kodierung des α-Galactosidase A Enzyms.
Kreul, Lukas
core   +1 more source