Results 61 to 70 of about 784,273 (205)

Mucopolysaccharidoses: clinical and epidemiological characteristics, cardiac manifestations, current approaches to diagnosis and treatment

Infusion & Chemotherapy
. This article presents a comprehensive analysis of the current scientific literature on mucopolysaccharidoses (MPS) ‒ a heterogeneous group of rare inherited lysosomal storage disorders.
P.F. Dudka, D.V. Dobrianskyi, D.I. Skorokhoda, V.V. Vozniuk, I.P. Tarchenko, L.I. Sokolova   +5 more
doaj   +1 more source

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

Conditions for safe and effective ADEPT treatment

, 2010
Antibody directed enzyme prodrug therapy (ADEPT) is a drug delivery system developed for the treatment of cancer. ADEPT uses a systemically administered antibody, tethered to an enzyme, to localize enzyme in tumour deposits.
Wilkins, D.K.
core  

Enzyme Replacement Therapy

, 2018
Enzyme replacement therapy is a therapeutic approach in which the specific enzyme that is absent or inactive in affected individuals is replaced with a functional enzyme molecule derived from biological sources or produced by biotechnology.
Abderrezak Khelfi
core   +1 more source

Combined cross-sectional prospective study to identify barriers to adherence of pancreatic enzyme use in patients with cystic fibrosis

, 2007
Monitoring and adjusting dose requirements of pancreatic enzyme replacement therapy (PERT) are an integral part of the dietetic assessment of patients with CF.
Pearson, Clare Emma
core  

Pregnancy is associated with elevation of liver enzymes in HIV-positive women on antiretroviral therapy.

, 2015
The objective of this study is to assess whether pregnancy is associated with an increased risk of liver enzyme elevation (LEE) and severe LEE in HIV-positive women on antiretroviral therapy (ART)
the UK Collaborative HIV Cohort (UK CHIC) Study, the UK, Ireland National Study of HIV in Pregnancy, Childhood (NSHPC),, Sabin, C, Thorne, C, Pillay, D, Huntington, S, Tookey, PA, Newell, ML, Taylor, GP, Anderson, J, Hill, T   +9 more
core  

Enzyme-Instructed Activation of Pro-protein Therapeutics In Vivo

, 2019
The selective and temporal control of protein activity in living cells provides a powerful tool to manipulate cellular function and to develop pro-protein therapeutics (PPT) for targeted therapy.
Qiao Tang (281623), Ming Wang (172316), Lanqun Mao (1430338), Weiqi Cai (7513556), Ying Jiang (5910), Jin Chang (1372593), Chunjing Liang (7513559), Xianghan Chen (7513562)   +7 more
core   +1 more source

Sapropterin hydrochloride: enzyme enhancement therapy for phenylketonuria

Therapeutic Advances in Endocrinology and Metabolism, 2011
Phenylketonuria (PKU) is an inherited disorder of amino acid metabolism caused by deficiency of the enzyme phenylalanine hydroxylase (PAH). Historically PKU was a common genetic cause of severe learning difficulties and developmental delay, but with the ...
Robin Lachmann
doaj   +1 more source

Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice

, 2015
Gaucher disease is caused by an inherited deficiency of the enzyme glucosylceramidase. Due to the lack of a fully functional enzyme there is progressive build-up of the lipid component glucosylceramide. Insufficient glucosylceramidase activity results in
Schambach, Axel, Rothe, Michael, Mirzaian, Mina, Modlich, Ute, Dahl, Maria,, Månsson, Jan-Eric, Karlsson, Stefan,, Aerts, Johannes M, Ehinger, Mats,, Marques, André R A, Lund University., Doyle, Alexander,, Olsson, Karin,   +12 more
core   +2 more sources

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy

Haematologica, 2012
Background Mucopolysaccharidosis type I is caused by deficiency of α-L-iduronidase. Currently available treatment options include an allogeneic hematopoietic stem cell transplant and enzyme replacement therapy.
Muhammad Ameer Saif, Brian W. Bigger, Karen E Brookes, Jean Mercer, Karen L. Tylee, Heather J. Church, Denise K. Bonney, Simon Jones, J. Ed Wraith, Robert F. Wynn   +9 more
doaj   +1 more source