Enzyme replacement therapy: efficacy and limitations

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Annotation Summary

Term Occurence Count Dictionary
mucopolysaccharidosis 1 endocrinologydiseases
Laronidase 2 endocrinologydiseasesdrugs
Sly syndrome 1 endocrinologydiseases
bortezomib 1 endocrinologydiseasesdrugs
dexamethasone 1 endocrinologydiseasesdrugs
lysosomal storage disease 2 endocrinologydiseases

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Select Drug Character Offset Drug Term Instance
Laronidase 4766 sulphateDermatan sulphate and heparan sulphateKeratan sulphate and chondroitin-6-sulphateDermatan sulphateDrug Laronidase (Aldurazyme®; Genzyme Europe B.V., Gooimeer 10, NL-1411 DD Naarden, The Netherlands), available since
Laronidase 27456 receptor, and therefore the mean plasma half-life of distinct ERT, has been recently hypothesized [[86]]. Laronidase has a mean plasma half-life ranging from 1.5 to 3.6 h, while idursulphase, galsulphase, and elosulphase
bortezomib 28555 titre and limited clinical efficacy of idursulphase treatment. Over 18 months, therapy with atumumab, bortezomib , methotrexate, short-term dexamethasone, and IVIG resulted in a significant reduction in neutralizing
dexamethasone 28592 idursulphase treatment. Over 18 months, therapy with atumumab, bortezomib, methotrexate, short-term dexamethasone , and IVIG resulted in a significant reduction in neutralizing anti-idursulphase IgG titre and a moderate
Select Disease Character Offset Disease Term Instance
Sly syndrome 3849 (Table 1). Recently, the recombinant enzyme ╬▓-glucuronidase has been tested for patients with MPS VII ( Sly syndrome ) [[4], [5]] and, to date, the treatment is available for commercial use in the United States where it
lysosomal storage disease 21016 MPS I and II.CNSIt is generally accepted that all the intravenous ERTs developed for MPS and other lysosomal storage disease s do not reach the CNS in amounts sufficient to prevent deterioration of CNS and neurocognitive functions
lysosomal storage disease 23203 withdraw will then be discussed with the family [[38]].In summary, ERTs developed for MPS and other lysosomal storage disease s do not reach the CNS in amounts sufficient to prevent deterioration of the CNS and neurocognitive function.Safety
mucopolysaccharidosis 301 11/2018Publication date (collection): /2018AbstractEnzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II, MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and

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