P. Harmatz | Semantic Scholar
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).. *P. Harmatz, C. Whitley, +7 authors. J. ... The important management topics are discussed in this review, and the use of enzyme-replacement therapy with recombinant human ... A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) ... Purpose: This study evaluated the safety and effectiveness of long-term enzyme replacement therapy with idursulfase ( ...
PDF] Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis | Semantic Scholar
... including experimental enzyme replacement therapy, gene therapy, cell-based therapy, and immunomodulation therapy were ... in modelling NCL disease pathology in various different model systems and their relevance to testing preclinical therapies so ...
7&agr;-Methyl-ethinyl estradiol is not a metabolite of tibolone but a chemical stress artifact | Semantic Scholar
Treatment with hormone replacement therapy (estrogens+progestogens), as well as with tibolone, is most effective in protecting ... Tibolone and its metabolites are not likely to play a clinically significant role at the level of these cytochrome P450 enzymes ... Investigation of whether tibolone, which is orally used for hormone replacement therapy, is transformed to a derivative of ... The role exerted by different progestins, currently used for contraception or in postmenopausal hormone replacement therapies, ...