Results 281 to 290 of about 460,260 (336)

Pancreatic enzyme replacement therapy

Current Gastroenterology Reports, 2001
Malabsorption due to severe pancreatic exocrine insufficiency is one of the most important late features of chronic pancreatitis. Generally, steatorrhea is more severe and occurs several years prior to malabsorption of other nutrients because synthesis and secretion of lipase are impaired more rapidly, its intraluminal survival is shorter, and the lack
P, Layer, J, Keller, P G, Lankisch
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Ten years of enzyme replacement therapy in paediatric onset mucopolysaccharidosis II in England.

Molecular Genetics and Metabolism, 2020
The outcome of 110 patients with paediatric onset mucopolysaccharidosis II (MPS II) since the commercial introduction of enzyme replacement therapy (ERT) in England in 2007 is reported. Median length of follow up was 10 years 3 months (range = 1 y 2 m to
A. Broomfield, J. Davison, Jane Roberts, C. Stewart, P. Hensman, C. Beesley, K. Tylee, S. Rust, B. Schwahn, E. Jameson, S. Vijay, S. Santra, S. Sreekantam, U. Ramaswami, A. Chakrapani, J. Raiman, M. Cleary, Simon A. Jones   +17 more
semanticscholar   +1 more source

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
exaly   +2 more sources

Enzyme Replacement Therapy for the Sphingolipidoses

1976
The greatest progress in the field of inheritable disorders during the past decade was made in the understanding and control of lipid storage diseases. Since original demonstrations in 1965 and 1966 of the metabolic defects in Gaucher’s disease (6,7) Niemann-Pick disease (8), Fabry’s disease (9), and metachromatic leukodystrophy (17), specific enzyme ...
R O, Brady, P G, Pentchev, A E, Gal, S R, Hibbert, J M, Quirk, G E, Mook, J W, Kusiak, J F, Tallman, A S, Dekaban   +8 more
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Enzyme-Replacement Therapy in Mucopolysaccharidosis I

New England Journal of Medicine, 2001
Mucopolysaccharidosis I is a lysosomal storage disease caused by a deficiency of the enzyme alpha-L-iduronidase. We evaluated the effect of enzyme-replacement therapy with recombinant human alpha-L-iduronidase in patients with this disorder.We treated 10 patients with mucopolysaccharidosis I (age, 5 to 22 years) with recombinant human alpha-L ...
Emil D. Kakkis, Joseph Muenzer, George E. Tiller, Lewis Waber, John Belmont, Merry Passage, Barbara Izykowski, Jeffrey Phillips, Robin Doroshow, Irv Walot, Richard Hoft, Kian Ti Yu, Susie Okazaki, Dave Lewis, Ralph Lachman, Jerry N. Thompson, Elizabeth F. Neufeld   +16 more
openaire   +2 more sources

Enzyme Replacement Therapy of Fabry Disease

Molecular Neurobiology, 2005
Fabry disease is an X-linked lysosomal storage disease caused by deficiency of the enzyme alpha-galactosidase A and results in pain, progressive renal impairment, cardiomyopathy, and cerebrovascular disease. The results of two major randomized, double-blind, placebo-controlled clinical trials and open-label extensions have shown that replacement of the
Joe T R, Clarke, R Mark, Iwanochko
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Fabry disease: enzyme replacement therapy

Journal of the European Academy of Dermatology and Venereology, 2003
ABSTRACTFabry disease is a multisystem disorder associated with wide variability in clinical expression. Fabry disease is an X‐linked lysosomal storage disorder caused by a deficiency of α‐galactosidase A. The enzyme defect leads to the systemic accumulation of glycosphingolipids with α‐galactosyl moieties consisting predominantly of ...
M R, Bongiorno, G, Pistone, M, Aricò
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Recombinant Enzyme Replacement Therapy in Hypophosphatasia

2015
Hypophosphatasia (HPP) is a rare monogenetic and multisystemic disease with involvement of different organs, including bone, muscle, kidney, lung, gastrointestinal tract and the nervous system. The exact metabolic mechanisms of the effects of TNAP deficiency in different tissues are not understood in detail.
Christine, Hofmann, Franz, Jakob, Lothar, Seefried, Birgit, Mentrup, Stephanie, Graser, Horacio, Plotkin, Hermann J, Girschick, Johannes, Liese   +7 more
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Enzyme-replacement therapy: Problems and prospects

Pharmaceutisch Weekblad Scientific Edition, 1989
Several diseases can, at least in theory, be treated by the administration of an enzyme, the deficiency of which is the cause of the disease. Various attempts have been made to correct enzymatic deficiencies responsible for the clinical manifestation of diseases for which prevention cannot be achieved by modification of the diet or by supportive ...
B, Rademaker, J, Raber
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Enzyme replacement therapy

Trends in Biochemical Sciences, 1981
ABSTRACT: Of the many genetic diseases which are now known to be due to an enzyme deficiency, the glycolipid storage diseases are among those which are most likely to yield to enzyme replacement therapy. To be successful, such therapy requires the availability of relatively pure enzyme from a human source, delivery of the enzyme to the storage cell ...
openaire   +1 more source