Results 31 to 40 of about 3,547,362 (239)

Enzyme Replacement Therapy and Fabry Nephropathy [PDF]

Clinical Journal of the American Society of Nephrology, 2010
Involvement of the kidneys in Fabry disease ("nephropathy") occurs in male and female individuals. The majority of patients with progressive nephropathy will have significant proteinuria and develop progressive loss of kidney function, leading to ESRD.
David G, Warnock, Erica, Daina, Giuseppe, Remuzzi, Michael, West   +3 more
openaire   +2 more sources

Reversal of life-threatening hepatopulmonary syndrome in Gaucher disease by imiglucerase enzyme replacement therapy

Molecular Genetics and Metabolism Reports, 2019
Advanced liver disease complicated by hepatopulmonary syndrome is a recognized complication of Gaucher disease. Macrophage-targeted, recombinant enzyme replacement therapy is effective in reversing clinical manifestations attributed to the accumulation ...
Amal El Beshlawy, Vagishwari Murugesan, Pramod Kumar Mistry, Khaled Eid   +3 more
doaj   +1 more source

Comparison of siRNA-mediated silencing of glycosaminoglycan synthesis genes and enzyme replacement therapy for mucopolysaccharidosis in cell culture studies [PDF]

, 2012
Cytotoxicity of laronidase (Aldurazyme®), employed in enzyme replacement therapy (ERT) for mucopolysaccharidosis type I (MPS I) and various siRNAs, tested previously in studies on substrate reduction therapy (SRT) for mucopolysaccharidoses, was ...
Banecka-Majkutewicz, Zyta, Węgrzyn, Alicja, Gabig-Cimińska, Magdalena, Jakóbkiewicz-Banecka, Joanna, Chmielarz, Izabela, Malinowska, Marcelina   +5 more
core   +2 more sources

Pancreatic Enzyme Replacement Therapy in hyperglycemic Male Rats

Kufa Journal for Veterinary Medical Sciences, 2020
The study was designed to evaluated exocrine pancreatic enzyme replacement therapy in hyperglycemic male rats induced by alloxan. A total of forty five adult male rats were use in this study, hyperglycemia was induce in thirty rats by single ...
Muhammad Ali Hameed Jalil, Nabeel M. N. Al-Sharafi   +1 more
doaj   +1 more source

Enzyme Replacement Therapy for Murine Hypophosphatasia [PDF]

Journal of Bone and Mineral Research, 2008
Abstract Introduction: Hypophosphatasia (HPP) is the inborn error of metabolism that features rickets or osteomalacia caused by loss-of-function mutation(s) within the gene that encodes the tissue-nonspecific isozyme of alkaline phosphatase (TNALP).
José Luis, Millán, Sonoko, Narisawa, Isabelle, Lemire, Thomas P, Loisel, Guy, Boileau, Pierre, Leonard, Svetlana, Gramatikova, Robert, Terkeltaub, Nancy Pleshko, Camacho, Marc D, McKee, Philippe, Crine, Michael P, Whyte   +11 more
openaire   +2 more sources

Study of enzyme replacement therapy for Gaucher Disease: comparative analysis of clinical and laboratory parameters at diagnosis and after two, five and ten years of treatment

Revista Brasileira de Hematologia e Hemoterapia, 2014
Objective: To evaluate the impact of enzyme replacement therapy for Gaucher Disease on clinical and laboratory parameters after two, five and ten years of treatment. Methods: Data were collected from patient records and analyzed using BioEstat software
Ana Maria Almeida Souza, Thiago Pimentel Muniz, Rafael Maciel Brito   +2 more
doaj   +1 more source

Randomised controlled trial of an interactive multimedia decision aid on hormone replacement therapy in primary care [PDF]

, 2001
Objective To determine whether a decision aid on hormone replacement therapy influences decision making and health outcomes.Design Randomised controlled trial.Setting 26 general practices in the United Kingdom.Participants 205 women considering hormone ...
Davis, H, Haines, A, Coulter, A, Gray, A, Murray, E, Tai, SS   +5 more
core   +1 more source

Enzyme replacement therapy for Gaucher disease [PDF]

Blood, 1991
Four patients with moderately severe type I Gaucher disease were treated with commercially available mannose terminated glucocerebrosidase (Ceredase; Genzyme, Boston, MA) for up to 13 months. The enzyme was administered at the rate of three to four times weekly at one fourth the total recommended dosage, greatly decreasing the cost.
E, Beutler, A, Kay, A, Saven, P, Garver, D, Thurston, A, Dawson, B, Rosenbloom   +6 more
openaire   +3 more sources

Enzyme Replacement Therapy in the Treatment of Fabry Disease

罕见病研究, 2023
Fabry disease is an X-linked inherited lysosomal storage disease caused by the mutation of GLA gene that encodes α-galactosidase A (α-Gal A). GLA gene mutation causes the decline or deficiency in the activity of α-Gal A, leading to the accumulation of ...
YU Che, WANG Rong
doaj   +1 more source

Polymeric nanoreactors for enzyme replacement therapy of MNGIE [PDF]

Journal of Controlled Release, 2010
The lack of a crucial metabolic enzyme can lead to accumulating substrate concentrations in the bloodstream and severe human enzyme deficiency diseases. Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) is such a fatal genetic disorder, caused by a thymidine phosphorylase deficiency.
De Vocht, Caroline, Ranquin, An, Van Ginderachter, Jo, Vanhaecke, Tamara, Rogiers, Vera, Van Gelder, Patrick, Versees, Wim, Steyaert, Jan   +7 more
openaire   +4 more sources