AAV |
Adeno-associated virus |
ADL |
Activity of daily living |
BBB |
Blood-brain barrier |
BMD |
Bone mineral density |
BMT |
Bone marrow transplantation |
C6S |
Chondroitin-6-sulfate |
CBA |
Chicken β-actin |
CSF |
Cerebrospinal fluid |
CT |
Computed tomography |
D8 |
Aspartic acid octapeptide |
DBS |
Dried blood spot |
DMB |
Dimethylmethylene blue |
ECM |
Extracellular matrix |
ELISA |
Enzyme-linked immunosorbent assay |
EMA |
European Medicines Agency |
ERT |
Enzyme replacement therapy |
FDA |
Food and Drug Administration |
FEV1 |
Forced expiratory volume in 1s |
FVC |
Forced vital capacity |
GAG |
Glycosaminoglycans |
GALNS |
N-acetylgalactosamine 6-sulfate sulfatase |
GVHD |
Graft versus host disease |
HS |
Heparan sulfate |
HSCT |
Hematopoietic stem cell transplantation |
KS |
Keratan sulfate |
LC-MS/MS |
Liquid chromatography tandem mass spectrometry |
MPS |
Mucopolysaccharidosis |
MRI |
Magnetic resonance imaging |
NLC |
Nanostructured lipid carriers |
SDET |
Substrate degradation enzyme therapy |