Results 71 to 80 of about 167,688 (127)

The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process. [PDF]

Drugs R D, 2021
Khan A, Sirrs SM, Bichet DG, Morel CF, Tocoian A, Lan L, West ML, Canadian Fabry Disease Initiative.   +7 more
europepmc   +1 more source

Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease

Biologics: Targets & Therapy, 2008
Fernando C Fervenza1, Roser Torra2, David G Warnock31Division of Nephrology and Hypertension, Mayo Clinic College of Medicine, Rochester, MN, USA; 2Department of Nephrology, Fundació Puigvert, Barcelona, Spain; 3Division of Nephrology ...
Fernando C Fervenza, Roser Torra, David G Warnock   +2 more
doaj  

Reducing agalsidase beta infusion time in Fabry patients: low incidence of antibody formation and infusion-associated reactions in an Italian multicenter study

Orphanet Journal of Rare Diseases
Background Fabry disease is a rare progressive X-linked lysosomal storage disease caused by mutations in the GLA gene that encodes α-galactosidase A. Agalsidase beta is a recombinant enzyme replacement therapy authorized in Europe at a standard dose of 1.
Renzo Mignani, Claudio Americo, Filippo Aucella, Yuri Battaglia, Vittoria Cianci, Annamaria Sapuppo, Chiara Lanzillo, Fabio Pennacchiotti, Luciano Tartaglia, Giacomo Marchi, Federico Pieruzzi   +10 more
doaj   +1 more source

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

, 2017
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 ...
West, Michael L., Beck, Michael, Kampmann, Christoph, Giugliani, Roberto, Ramaswami, Uma, Hughes, Derralynn A., Pintos-Morell, G.   +6 more
core  

Enzyme replacement therapy in Fabry Disease: the importance of dose

Revista de Nefrología, Diálisis y Trasplante, 2017
Fabry disease is a rare X-linked inherited disorder due to deficient or absent lysosomal α-galactosidase A activity, resulting in an excessive glycosphingolipid deposit, mainly globotriaosylceramide (gl3) and mortality due renal, cardiac and neurological
Juan Manuel Politei, Andrea B. Schenone, Consuelo Durand, Alberto Ortiz   +3 more
doaj  

A systematic literature review on the health-related quality of life and economic burden of Fabry disease

Orphanet Journal of Rare Diseases
Background Fabry disease (FD) is a rare lysosomal storage disease associated with glycolipid accumulation that impacts multiple physiological systems. We conducted a systematic literature review (SLR) to characterize the humanistic (quality of life [QoL])
Ana Jovanovic, Eve Miller-Hodges, Felicia Castriota, Shweta Takyar, Heena Howitt, Olulade Ayodele   +5 more
doaj   +1 more source

New Drugs Available for Fabry Disease

Kidney & Blood Pressure Research
Background: Fabry disease (FD) is an X-linked genetic condition caused by variants in the GLA gene, leading to a deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A) and the accumulation of complex glycosphingolipids such as ...
Fernando Perretta, Gustavo Cabrera, Juan Politei   +2 more
doaj   +1 more source

Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease

Drug Design, Development and Therapy, 2015
Lubor Goláň,1 Ozlem Goker-Alpan,2 Myrl Holida,3 Ikka Kantola,4 Mariusz Klopotowski,5 Johanna Kuusisto,6 Aleš Linhart,1 Jacek Musial,7 Kathleen Nicholls,8 Derlis Gonzalez-Rodriguez,9 Reena Sharma,10 Bojan Vujkovac,11 Peter Chang,12 Anna ...
Goláň L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A   +13 more
doaj  

Analisi molecolare con Risonanza Magnetica Nucleare del tessuto miocardico in pazienti affetti da malattia di Anderson-Fabry: valore aggiunto della Risonanza Magnetica Cardiaca nell'identificazione e nel follow-up dei pazienti affetti da malattia di Anderson-Fabry in trattamento con enzima ricombinante agalsidase beta a dopo "switch therapy" con agalsidase alfa [PDF]

, 2013
Scopo La malattia di Anderson-Fabry è determinata da un disordine multi-sistemico del metabolismo lipidico dovuta alla carenza dell’enzima idrolitico α-galactosidase A (α-Gal A), secondario ad una alterazione del cromosoma X, che provoca un accumulo di ...
Messalli, Giancarlo
core   +1 more source

A incorporação de tecnologias em saúde para doenças raras no SUS

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução: Desde a vigência da Política Nacional de Doenças Raras, publicada pela Portaria GM/MS 199/2014 observa-se um crescente interesse do Ministério da Saúde do Brasil na estruturação de sua rede de cuidado no campo das doenças raras, bem como na ...
Tacila Pires Mega, Artur Felipe Siqueira de Brito   +1 more
doaj   +1 more source