Table 3.
Somatic response on enzyme replacement therapy
| Sign/symptom | Improvement | Stabilization | Disease progression |
|---|---|---|---|
| Liver and/or spleen sizea,b | 22/22 (100 %) | 0 | 0 |
| Frequency of respiratory infectionsc | 22/22 (100 %) | 0 | 0 |
| Texture of hair and/or skina | 21/21 (100 %) | 0 | 0 |
| Disease-related hospitalizations | 11/14 (79 %) | 3/14 (21 %) | 0 |
| Diarrheac | 4/9 (44 %) | 5/9 (56 %) | 0 |
| Joint range of motion (1 or more joints)a | 7/21 (33 %) | 13/21 (62 %) | 1/21 (5 %) |
| Skeletal diseaseb | 2/22 (9 %) | 19/22 (86 %) | 1/22 (5 %) |
| Cardiac diseased | 1/22 (5 %) | 19/22 (86 %) | 2/22 (9 %) |
aAssessed by physical exam
bAssessed by imaging studies
cAssessed by caregiver report
dAssessed by electrocardiogram and/or echocardiogram