MPS subtype | Number of pts (male; female) | Clinical phenotype | Age at diagnosis (years)§ | Time between diagnosis and start of ERT (years)§ | Age at start of ERT (years)§ |
---|---|---|---|---|---|
MPS I | 9 (6 M; 3F) | 6 non-neuropathic, 3 neuropathic | 2.5 ± 2# | 1.5 ± 1.8 | 6.2 ± 6 |
MPS II | 16 (15 M; 1F) | 6 non-neuropathic, 11 neuropathic | 4.6 ± 3* | 4.6 ± 8 | 10.5 ± 10* |
MPS IIIA | 8 (5 M; 3F) | 1 mild, 7 severe | 6.27 ± 5 | – | – |
MPS IIIB | 5 (2 M;3F) | 5 severe | 4.28 ± 3 | – | – |
MPS IIID | 1 (1 M; 0F) | 1 severe | 5.48 | – | – |
MPS IVA | 12 (4 M; 8F) | 5 slowly progressing,7 rapidly progressing | 3.3 ± 2 | 9.5 ± 9.9 | 12.8 ± 10 |
MPS IVB | 2 (2 M; 0F) | 2 slowly progressing | 10 ± 3 | – | – |
MPS VI | 7 (2 M; 5F) | 2 slowly progressing, 5 rapidly progressing | 2 ± 0.7 | 4.7 ± 3 | 6.7 ± 3 |