Results 31 to 40 of about 784,273 (205)
Case Report: Mucopolysaccharidosis Type I Treatment With α-L-Iduronidase Replacement Therapy
Frontiers in Pediatrics, 2022 Mucopolysaccharidosis is a rare disease and can be divided into seven different subtypes, according to the affected enzyme. Mucopolysaccharidosis type I, the first subtype discovered and reported, mainly affects the in vivo storage of degraded sugar. The
Ying Li, Deyun Liu, Yue Yu
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Trends in Enzyme Therapy for Phenylketonuria
Molecular Therapy, 2004 Phenylketonuria (PKU) is an inborn error of amino acid metabolism caused by phenylalanine hydroxylase (PAH) deficiency. Dietary treatment has been the cornerstone for controlling systemic phenylalanine (Phe) levels in PKU for the past 4 decades. Over the years, it has become clear that blood Phe concentration needs to be controlled for the life of the ...
Kim, Woomi +7 more
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Onset of experimental severe cardiac fibrosis is mediated by overexpression of angiotensin-converting enzyme 2 [PDF]
, 2009 Angiotensin-converting enzyme (ACE) 2 is a recently identified homologue of ACE. There is great interest in the therapeutic benefit for ACE2 overexpression in the heart.
Smith, Godfrey +12 more
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, 2022 Lysosomal acid lipase deficiency (LAL-D) presents as one of two rare autosomal recessive diseases: Wolman disease (WD), a severe disorder presenting in infancy characterized by absent or very low LAL activity, and cholesteryl ester storage disease (CESD),
Ashbrook, Anna +11 more
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Molecular Genetics and Metabolism Reports, 2021 GM1-gangliosidosis is a lysosomal disease resulting from a deficiency in the hydrolase β-galactosidase (β-gal) and subsequent accumulation of gangliosides, primarily in neuronal tissue, leading to progressive neurological deterioration and eventually ...
Michael J. Przybilla +10 more
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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 +5 more
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Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis [PDF]
, 2019 Agalsidasa alfa; Teràpia de reemplaçament enzimàtic; Malaltia de FabryAgalsidasa alfa; Terapia de reemplazo de enzimas; Enfermedad de FabryAgalsidase alfa; Enzyme replacement therapy; Fabry diseasePurpose: Following the publication of 5-year agalsidase ...
Beck, Michael +5 more
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Chronic pancreatitis: general principles of treatment
Gastroenterologìa, 2018 The literature review considers the problems of the treatment of abdominal pain syndrome, pancreatic exocrine insufficiency syndrome, chronic pancreatitis of Oddi’s sphincter dysfunction, disorders of gall bladder, bile ducts, dysbiosis, intestinal ...
T.N. Hristich, D.O. Hontsariuk
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Advancing Therapeutic Strategies for Celiac Disease: From Gluten-Free Diets to Novel Biopharmaceutical Approaches [PDF]
BIO Web of ConferencesGluten is a type of protein primarily present in wheat, barley and rye, which contributes towards the texture and elasticity of bread and other baking products.
Aindree, Prakash Singh Rajnish
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, 2019 Mesoporous silica nanoparticles (MSNs) have been proved potential nanocarriers for efficient stimuli-triggered drug delivery systems (DDSs) in cancer therapy.
Yuan Liu (88411) +5 more
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