Results 31 to 40 of about 498,682 (384)

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

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy. [PDF]

, 2016
BackgroundFollowing a drug manufacturing process change, safety/efficacy of agalsidase alfa were evaluated in enzyme replacement therapy (ERT)-naïve children with Fabry disease.MethodsIn an open-label, multicenter, Phase II study (HGT-REP-084; Shire), 14
Chang, Peter, Goker-Alpan, Ozlem, Longo, Nicola, McDonald, Marie, Pano, Arian, Schiffmann, Raphael, Shankar, Suma P, Shen, Yinghua   +7 more
core   +2 more sources

The use of pancreatic enzyme replacement therapy in patients with a diagnosis of chronic pancreatitis and pancreatic cancer in the US is infrequent and inconsistent

Alimentary Pharmacology and Therapeutics, 2020
Patients with chronic pancreatitis or pancreatic cancer commonly develop exocrine pancreatic insufficiency, and may not be adequately treated with pancreatic enzyme replacement therapy (PERT).
C. Forsmark, Gong Tang, Hongzhi Xu, M. Tuft, S. Hughes, D. Yadav   +5 more
semanticscholar   +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.
P. Garver, Andrea C. Kay, Ernest Beutler, Arthur Dawson, D. Thurston, Alan Saven, Barry E. Rosenbloom   +6 more
openaire   +4 more sources

Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy

Therapeutics and Clinical Risk Management, 2020
Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a progressive neurodegenerative disease that results in early-onset, severe, progressive, neurological disabilities, leading to death in late childhood or early adolescence. Management has relied on
N. Specchio, N. Pietrafusa, M. Trivisano
semanticscholar   +1 more source

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease [PDF]

, 2014
Background: Infantile Pompe disease is a rare metabolic disease. Patients generally do not survive the first year of life. Enzyme replacement therapy (ERT) has proven to have substantial effects on survival in infantile Pompe disease.
Hakkaart-van Roijen, L. (Leona), Hoogenboom-Plug, I (Iris), Kanters, T.A. (Tim), Ploeg, A.T. (Ans) van der, Redekop, W.K. (Ken), Rutten-van Mölken, M.P.M.H. (Maureen)   +5 more
core   +2 more sources

Engineering of GlcNAc-1-phosphotransferase for production of highly phosphorylated lysosomal enzymes for enzyme replacement therapy [PDF]

, 2017
Several lysosomal enzymes currently used for enzyme replacement therapy in patients with lysosomal storage diseases contain very low levels of mannose 6-phosphate, limiting their uptake via mannose 6-phosphate receptors on the surface of the deficient ...
Doray, Balraj, Kornfeld, Stuart, Lee, Wang-Sik, Liu, Lin   +3 more
core   +3 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

Development of the “Hamburg Best Practice Guidelines for ICV−Enzyme Replacement therapy (ERT) in CLN2 Disease” Based on 6 Years Treatment Experience in 48 Patients

Neuropediatrics, 2020
Intracerebroventricular enzyme replacement therapy (ICV-ERT) for CLN2 disease represents the first approved treatment for neuronal ceroid lipofuscinosis (NCL) diseases.
C. Schwering, G. Kammler, E. Wibbeler, M. Christner, J. Knobloch, M. Nickel, J. Denecke, M. Baehr, A. Schulz   +8 more
semanticscholar   +1 more source

Clinical outcome of a patient with lysosomal acid lipase deficiency and first results after initiation of treatment with Sebelipase alfa: A case report [PDF]

, 2019
We report on a case of very rare autosomal recessive cholesteryl ester storage disease due to lysosomal acid lipase deficiency (LALD). LALD is caused by mutations in the lysosomal acid lipase A (LIPA) gene resulting in cholesteryl ester accumulation in
Bobbert, Thomas, Demuth, Ilja, Haberbosch, Linus, Hollstein, Tim, Kassner, Ursula, Soll, Dominik, Spira, Dominik, Spranger, Joachim, Steinhagen-Thiessen, Elisabeth   +8 more
core   +1 more source