| MPS II | Mucopolysaccharidosis type II |
| NBS | Newborn screening |
| ICV | Intracerebroventricular |
| ERT | Enzyme replacement therapy |
| ICV-ERT | Intracerebroventricular enzyme replacement therapy |
| CBT | Cord blood transplantation |
| HS | Heparan sulfate |
| LSDs | Lysosomal storage disorder |
| I2S | Iduronate-2-sulfatase |
| GAGs | Glycosaminoglycans |
| CNS | Central nervous system |
| HSCT | Hematopoietic stem cell transplantation |
| BBB | Blood–brain barrier |
| 4-MU | 4-methylumbelliferone |
| MS/MS | Tandem mass spectrometry |
| GVHD | Graft-versus-host disease |
| CSF | Cerebrospinal fluid |
| DA | Developmental age |
| DQ | Developmental quotient |
| KSPD | Kyoto Scale of Psychological Development |
| SOS | Sinusoidal obstruction syndrome |
| IEM | Inborn errors of metabolism |