Specifically, odiparcil acts on chondroitin sulfate ("CS") and
dermatan sulfate ("DS"), either or both of which accumulate in patients with MPS I, II, IVa, VI and VII.
It was shown that loss of sulfated proteoglycans and anionic regions in the basal membrane of the glomerulus and the mesangial mat rix caused excess accumulation of proteoglycans such as chondroitin sulphate and
dermatan sulphate in these areas.3 This causes a decrease in charge-dependent renal selectivity and an increase in the thickness of the basal membrane.
Odiparcil has also the potential to address other MPS types, characterized by the accumulation of chondroitin or
dermatan sulfate (MPS I or Hurler/Sheie syndrome, MPS II or Hunter syndrome, MPS IVa or Morquio syndrome and MPS VII or Sly syndrome).
Hastalik, iduronat-2-sulfataz enzim eksikligi nedeniyle yikilamayan
dermatan ve heparan sulfat gibi glukoazminoglikanlarin (GAG) solunum, sinir, dolasim ve kas-iskelet sistemlerinde birikmesi ile karakterizedir.
Epidural fibrosis is a natural response of the body to the impact of surgery, leading to marked reduction in the cellularity of the tissue and excessive deposition of the extracellular matrix components such as collagen, fibronectin, and
dermatan sulfate.
60 kDa) (Rodrigues et al., 2013),
dermatan sulfate (DS) and/or heparan sulfate (HS) (Salles et al., 2017).
In Cohort 2 at 16 weeks post-dosing, mean reductions were observed in total urinary GAGs,
dermatan sulfate, and heparan sulfate of 51%, 32%, and 61%, respectively.
MPS II is a genetic lysosomal storage disease caused by deficiency of the iduronate-2-sulfatase enzyme which is needed to break down or recycle glycosaminoglycans
dermatan sulfate and heparan sulfate.
El acido hialuronico (AH) pertenece al grupo de los glucosaminoglucanos que son polisacaridos lineales, formados por un amino azucar y un acido uronico o galactosa (Kogan et al., 2007), como lo son el
dermatan sulfato, condroitina 4-sulfato y condroitina 6-sulfato (Kakehi et al., 2003), siendo el AH el mas simple en estructura, asi tambien el unico no asociado covalentemente con un nucleo proteico, ademas de que es sintetizado en la matriz extracelular del tejido conectivo al contrario de los demas glucosaminoglucanos que son sintetizados en el aparato de Golgi.
Decorin (DCN) is one of the most common
dermatan sulphate proteoglycan (molecular protein polysaccharide) with small leucine rich proteins.
Generally,
dermatan sulphate deposition in the MPS syndromes results cardiac valve problems.
Urine
dermatan sulphate concentrations were elevated in both of them and the gross elevations of plasma beta-hexosaminidase, arylsulphatase A and alphamannosidase were consistent with a diagnosis of ML II/III.
