Table 2 Caregiver-reported improvements in signs and symptoms and disease impact following participation in the idursulfase-IT triala
Improvement | Children in the pivotal trial and substudy N = 39 n (%) | Children in the substudy N = 6 n (%) |
|---|---|---|
Cognitive improvements | 37 (94.9) | 5 (83.3) |
Improved speech | 21 (53.8) | 4 (66.7) |
Improved ability to communicate needs or wants/engage in conversation | 20 (51.3) | 1 (16.7) |
Continuing to gain skills | 18 (46.2) | 5 (83.3) |
Improved recall and memory | 9 (23.1) | – |
Exhibiting higher-level thinking skills | 9 (23.1) | – |
Emotional/behavioral improvements | 26 (66.7) | 1 (16.7) |
Improved ability to focus/longer attention span | 13 (33.3) | 1 (16.7) |
Decreased aggression | 12 (30.8) | 1 (16.7) |
Decreased hyperactivity | 8 (20.5) | – |
Improved mood stability | 2 (5.1) | – |
Improvements in activities of daily living | 22 (56.4) | 3 (50.0) |
Improved potty training | 13 (33.3) | 2 (33.3) |
Improved ability to dress oneself | 8 (20.5) | 3 (50.0) |
Improved ability to bathe oneself or brush one’s teeth | 5 (12.8) | 1 (16.7) |
Improved ability to feed oneself | 2 (5.1) | – |
Social improvements | 17 (43.6) | – |
Physical improvements | 12 (30.8) | 2 (33.3) |
Improved balance | 6 (15.4) | 1 (16.7) |
Improved mobility/decreased stiffness | 5 (12.8) | 1 (16.7) |
Improved vision | 2 (5.1) | – |
Improvements in family dynamics | 4 (10.3) | – |