BBB |
Blood-Brain Barrier |
IDS |
Iduronate 2-Sulfatase (Elaprase™) |
GAG |
Glycosaminoglycan |
LC-MS/MS |
Liquid Chromatography-Tandem Mass Spectrometry |
HS |
Heparan Sulfate |
DS |
Dermatan Sulfate |
CSF |
Cerebrospinal Fluid |
TfR |
Transferrin Receptor |
MPS II |
Mucopolysaccharidosis II |
ETV |
Enzyme Transport Vehicle |
D0A0 |
4,5 unsaturated uronic acid residue-N-acetyl glucosamine, HS ∆UA-GluNAc |
D0S0 |
4,5 unsaturated uronic acid residue-glucosamine-N-sulfate, ∆UA-GluNS |
D0a4 |
4,5 unsaturated uronic acid residue-N-acetyl galactosamine, 4-O-sulfate, ∆UA-GalNAc4S |
HSCT |
Hematopoietic Stem Cell Transplant |
ERT |
Enzyme Replacement Therapy |
Nf-L |
Neurofilament Light Chain |
TfRmu/hu KI |
Chimeric human/mouse Transferrin Receptor Knock-In mouse |