Results 31 to 40 of about 133,872 (296)

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

Pancreatic Enzyme Replacement Therapy in Pancreatic Cancer [PDF]

Cancers, 2020
Pancreatic cancer is an aggressive malignancy and the seventh leading cause of global cancer deaths in industrialised countries. More than 80% of patients suffer from significant weight loss at diagnosis and over time tend to develop severe cachexia. A major cause of weight loss is malnutrition. Patients may experience pancreatic exocrine insufficiency
Raffaele Pezzilli, Riccardo Caccialanza, Gabriele Capurso, Oronzo Brunetti, Michele Milella, Massimo Falconi   +5 more
openaire   +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

Characterization and downstream mannose phosphorylation of human recombinant α-L-iduronidase produced in Arabidopsis complex glycan-deficient (cgl) seeds [PDF]

, 2013
This work was supported by a Wellcome Trust award to TMG.Mucopolysaccharidosis (MPS) I is a lysosomal storage disease caused by a deficiency of α-L-iduronidase (IDUA) (EC 3.2.1.76); enzyme replacement therapy is the conventional treatment for this ...
Brooks, Doug, Gloster, Tracey M., Vocadlo, D.J., Brooks, D., Kolarich, Daniel, Kermode, Allison R, Kermode, Allison R., Haselhorst, T., Qian, Y., Gloster, Tracey M, Qian, Yi, Gloster, Tracey, Kolarich, D., Haselhorst, Thomas, Packer, N.H., Pierce, Owen, He, X., von Itzstein, Mark, Gloster, T.M., Packer, Nicolle H., He, Xu, Pierce, O., Packer, Nicolle H, Vocadlo, David J, von Itzstein, M., Kermode, A.R., Vocadlo, David J.   +26 more
core   +1 more source

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

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

The COVID-19 Pandemic and Enzyme Replacement Therapy in Lysosomal Storage Disorders [PDF]

, 2021
Aim:The coronavirus disease-2019 (COVID-19) pandemic has caused a worldwide public health emergency, especially affecting people with chronic illnesses including lysosomal storage disorders (LSDs).
Çiğdem Seher Kasapkara, Kilic, Mustafa, Molla, Gulhan Karakaya, Yılmaz Yıldız, KASAPKARA, ÇİĞDEM SEHER, Burak Açıkel, Gülhan Karakaya Molla, Yildiz, Yilmaz, Acikel, Burak, Mustafa Kılıç, Olgaci, Asburce, Asburçe Olgaç   +11 more
core   +1 more source

Functional assessment using short tests in a patient with Pompe disease receiving enzyme replacement therapy: case report

Case Reports, 2019
Introduction: Pompe disease is characterized by the deficiency of the acid alfa glucosidase enzyme, which leads to a glycogen accumu­lation mainly in cardiac and skeletal muscles.
Thomas Torres-Cuenca, Jorge Arturo Díaz-Ruíz, Fernando Ortiz-Corredor   +2 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
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