Results 1 to 10 of about 790 (99)

Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy [PDF]

Molecular Genetics and Metabolism Reports
Background: Analyses of up to 20 years of data from the Fabry Outcome Survey (FOS) assessed the long-term effectiveness of agalsidase alfa enzyme replacement therapy.
Christina Anagnostopoulou, Christoph Kampmann   +1 more
exaly   +4 more sources

Updated Evaluation of Agalsidase Alfa Enzyme Replacement Therapy for Patients with Fabry Disease: Insights from Real-World Data [PDF]

Drug Design, Development and Therapy
Sandro Feriozzi,1 Cristina Chimenti,2 Ricardo Claudio Reisin3 1Department of Nephrology and Dialysis Unit, Belcolle Hospital Viterbo, Italy; 2Department of Clinical Sciences, Internal Medicine, Anesthesiology and Cardiovascular Sciences, La Sapienza ...
Cristina Chimenti, Sandro Feriozzi, Ricardo C Reisin   +2 more
exaly   +4 more sources

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 [PDF]

Orphanet Journal of Rare Diseases, 2023
Background Pegunigalsidase alfa is a novel, PEGylated α-galactosidase-A enzyme-replacement therapy approved in the EU and US to treat patients with Fabry disease (FD). Objective/methods BRIDGE is a phase 3 open-label, switch-over study designed to assess
Aleš Linhart, Gabriela Dostálová, Kathy Nicholls, Michael L. West, Camilla Tøndel, Ana Jovanovic, Pilar Giraldo, Bojan Vujkovac, Tarekegn Geberhiwot, Einat Brill-Almon, Sari Alon, Raul Chertkoff, Rossana Rocco, Derralynn Hughes   +13 more
doaj   +2 more sources

Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naïve Fabry disease patients [PDF]

BMC Pharmacology and Toxicology, 2017
Background Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the α-galactosidase A gene mutations. Enzyme-replacement-therapy (ERT) products for FD currently used include agalsidase alfa and agalsidase beta.
Kazuya Tsuboi, Hiroshi Yamamoto
doaj   +2 more sources

Long-term safety of enzyme replacement therapy with agalsidase alfa in patients with Fabry disease: post-marketing extension surveillance in Japan [PDF]

Molecular Genetics and Metabolism Reports
Fabry disease is a rare inherited X-linked metabolic disorder in which deficient alpha-galactosidase A activity causes progressive build-up of globotriaosylceramide (Gb3) and multi-system dysfunction.
Makoto Arakawa, Yoshinori Ikeda, Hiromichi Otaka, Sanghun Iwashiro   +3 more
doaj   +2 more sources

Clinical and Biochemical Improvement After Switching From Agalsidase Alfa to Beta in a Boy With Classic Fabry Disease: A Case Report [PDF]

Clinical Case Reports
We described a 12‐year‐old boy with classic Fabry disease who was diagnosed through newborn screening. At age 6.2, he started agalsidase alfa based on evidence of subclinical organ involvement. At age 7.2, acroparesthesia subsequently developed.
Nobuhiko Koga, Hiroaki Yodogawa, Takaaki Sawada, Yuichi Mushimoto, Shinichiro Nagamitsu, Kimitoshi Nakamura, Shinichi Hirose, Takahito Inoue   +7 more
doaj   +2 more sources

Sex-based antibody subclass maturation drives direct enzymatic inhibition in fabry disease patients receiving enzyme replacement therapy [PDF]

Frontiers in Molecular Biosciences
BackgroundEnzyme replacement therapy (ERT) for Fabry disease can elicit anti-drug antibodies (ADAs) that may diminish efficacy and limit clinical benefit.ObjectivesTo develop and apply a multiplexed proteomic assay for quantitative, subclass-specific ADA
Tomas Baldwin, Hibba Kurdi, Ivan Doykov, Francesca Robertson, Stefania Rosmini, Sabrina Nordin, Sabrina Nordin, Joao Augusto, Rebecca Kozor, Rebecca Kozor, Julien Baruteau, Julien Baruteau, James Davison, David Moreno-Martinez, Uma Ramaswami, Ravi Vijapurapu, Tarekegn Geberhiwot, Rick Steeds, James Moon, James Moon, Derralynn Hughes, Derralynn Hughes, Kevin Mills, Wendy E. Heywood   +23 more
doaj   +2 more sources

Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis [PDF]

Drug Design, Development and Therapy, 2021
Derralynn Hughes,1 Aleš Linhart,2 Andrey Gurevich,3 Vasiliki Kalampoki,3 Dalia Jazukeviciene,3 Sandro Feriozzi4 On behalf of the FOS Study Group1University College London and Royal Free London NHS Foundation Trust, London, UK; 2Charles University, First ...
Hughes D, Linhart A, Gurevich A, Kalampoki V, Jazukeviciene D, Feriozzi S   +5 more
doaj   +2 more sources

A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta [PDF]

Orphanet Journal of Rare Diseases
Background Fabry disease (FD) is a rare inherited X-linked lysosomal disorder caused by the deficiency or dysfunction of the enzyme α-galactosidase.
Ian Keyzor, Ana Maria Martins, Sema Kalkan Uçar, Hiroyuki Yamakawa, Yin-Hsiu Chien, Nur Arslan, Dau-Ming Niu, Leyla Tümer, Laura Baldock, Simon Shohet, Joseph D. Giuliano   +10 more
doaj   +2 more sources

Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events [PDF]

Molecular Genetics and Metabolism Reports, 2018
This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately ...
Michael Beck, Derralynn Hughes, Christoph Kampmann, Guillem Pintos-Morell, Uma Ramaswami, Michael L. West, Roberto Giugliani   +6 more
doaj   +2 more sources