Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future.

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Term Occurence Count Dictionary
Idursulfase 12 endocrinologydiseasesdrugs
lysosomal storage disease 2 endocrinologydiseases
mucopolysaccharidosis 5 endocrinologydiseases
sodium chloride 2 endocrinologydiseasesdrugs
Fabry disease 1 endocrinologydiseases

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Select Drug Character Offset Drug Term Instance
Idursulfase 7394 posttranslational modification profile is retained.[28]Box 1Key points in the development of idursulfase Idursulfase produced in a novel new protein production platform in a human HT-1080 fibrosarcoma cell line[21],[107]–[110]◦HT-1080
Idursulfase 7697 transformation event than the more commonly used CHO cell line and does not contain virus-like particles Idursulfase is structurally similar to endogenous enzyme[21]◦Expressed as a single 550 amino acid polypeptide
Idursulfase 8374 impact on immunological aspects[113] and on preventing rapid clearance of the enzyme from the body[113] Idursulfase has similar activity to endogenous enzyme[21]◦Same hydrolytic targets as endogenous enzyme (the 2-sulfate
Idursulfase 11866 clinical trials and laid a strong foundation for the design of the human clinical trial program.[21] Idursulfase clinical development programThe development of a therapy for any rare disease poses significant challenges;
Idursulfase 14921 on the results of this single, relatively small, pivotal trial (study design is given in Table 1). Idursulfase was well tolerated, and no patient withdrew owing to an IRR.[32] Compared with placebo, the greatest
Idursulfase 15632 II/III study, idursulfase 0.5 mg/kg weekly was approved in the USA in 2006 and in Europe in 2007.[32] Idursulfase post-approval programsA wealth of knowledge has been accumulated since idursulfase was first approved,
Idursulfase 39261 idursulfase for MPS II was a big step forward for patients and families living with this devastating disease. Idursulfase was the first therapy to specifically address the underlying cause of the disease and, in conjunction
Idursulfase 41086 this aspect of the disease.Figure 1Cellular uptake and intracellular trafficking of idursulfase.Notes: Idursulfase (red) is taken up by cells via M6P receptor (dark blue)-mediated endocytosis. The enzyme is then trafficked
Idursulfase 43272 double-blind, placebo-controlledII/III; open-label extensionIV; open-label, single-armTreatment regimen(s) Idursulfase 0.15 mg/kg, 0.5 mg/kg or 1.5 mg/kg EOWIdursulfase 0.5 mg/kg EOW or weeklyIdursulfase 0.5 mg/kg weeklyIdursulfase
Idursulfase 43322 extensionIV; open-label, single-armTreatment regimen(s)Idursulfase 0.15 mg/kg, 0.5 mg/kg or 1.5 mg/kg EOW Idursulfase 0.5 mg/kg EOW or weeklyIdursulfase 0.5 mg/kg weeklyIdursulfase 0.5 mg/kg weeklyStudy duration6 months5.5
Idursulfase 43357 single-armTreatment regimen(s)Idursulfase 0.15 mg/kg, 0.5 mg/kg or 1.5 mg/kg EOWIdursulfase 0.5 mg/kg EOW or weekly Idursulfase 0.5 mg/kg weeklyIdursulfase 0.5 mg/kg weeklyStudy duration6 months5.5 years12 months24 months12 monthsNumber
Idursulfase 43385 0.15 mg/kg, 0.5 mg/kg or 1.5 mg/kg EOWIdursulfase 0.5 mg/kg EOW or weeklyIdursulfase 0.5 mg/kg weekly Idursulfase 0.5 mg/kg weeklyStudy duration6 months5.5 years12 months24 months12 monthsNumber of patients1212969428Patient
sodium chloride 11384 extractable volume of 3.0 mL (the excipients are all commonly used in parenteral protein solutions: sodium chloride , a sodium phosphate buffer, polysorbate 20 and water for injections; pĤ6). Dilution prior to administration
sodium chloride 11524 buffer, polysorbate 20 and water for injections; pĤ6). Dilution prior to administration is in 0.9% USP sodium chloride injection.[21],[32],[51]Abbreviation: USP, US Pharmacopeial Convention.Overall, the results of this
Select Disease Character Offset Disease Term Instance
Fabry disease 12623 program built on experience gained in the development of ERTs for other LSDs (including Gaucher disease, Fabry disease and mucopolysaccharidosis type I [MPS I]) and comprised a Phase I/II study followed by a larger pivotal
lysosomal storage disease 456 8/2017AbstractMucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare, multisystemic, progressive lysosomal storage disease caused by deficient activity of the iduronate-2-sulfatase (I2S) enzyme. Accumulation of the glycosaminoglycans
lysosomal storage disease 2523 type II (MPS II; Hunter syndrome; OMIM 309900), one of a group of rare inherited disorders known as lysosomal storage disease s (LSDs), was first described in 1917.[1] LSDs are characterized by defects in the functioning of lysosomal
mucopolysaccharidosis 82 Title: Drug Design, Development and TherapyDevelopment of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the futureDavid AH WhitemanAlan KimuraResearch
mucopolysaccharidosis 12641 experience gained in the development of ERTs for other LSDs (including Gaucher disease, Fabry disease and mucopolysaccharidosis type I [MPS I]) and comprised a Phase I/II study followed by a larger pivotal Phase II/III study designed
mucopolysaccharidosis 19950 Clinical Understanding Scale; IRR, infusion-related reaction; LVMI, left ventricular mass index; MPS II, mucopolysaccharidosis type II; SAE, serious AE; uGAG, urinary glycosaminoglycan.Overall, the registry contains data from more
mucopolysaccharidosis 37947 technologies.[96]–[99] Although viral vector gene therapy approaches have reached clinical trials for MPS I and mucopolysaccharidosis type III (MPS III), the technology remains at a preclinical stage for MPS II. Finally, various compounds
mucopolysaccharidosis 46596 forced expiratory volume in 1 second; FVC, forced vital capacity; I2S, iduronate-2-sulfatase; MPS II, mucopolysaccharidosis type II; uGAG, urinary glycosaminoglycan

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