Table 1 Characteristics of the participants
From: Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study
Gaucher | Fabry | MPS III | ||
|---|---|---|---|---|
Patients | Parents# | |||
Demographics | ||||
Number | 9 | 23 | 2 | 5 |
Female sex (n) | 3 | 17 | 0 | 3 |
Age (median, range) | 59 (49–72) | 50 (20–64) | 20,23 | 10 (18m–26y) |
Current treatment | ||||
ERT (n) | 9 | 17 | 0 | 0 |
SRT (n) | 0 | 1 | 0 | 0 |
Investigational treatment (Clinical trial) (n) | 0 | 1 | 0 | 2 |
No disease specific treatment (n) | 0 | 4 | 2 | 3 |
Disease subtype | ||||
Gaucher type 1 (n) | 9 | – | – | – |
Classical Fabry disease (n) | – | 19 | – | – |
Non-classical Fabry disease (n) | – | 4 | – | – |
MPS IIIA (n) | – | – | 2 | 4 |
MPS IIIC (n) | – | – | 0 | 1 |