Table 5 History of NP-C manifestations for patients continuously treated with miglustat
Manifestation | N | n (%) |
|---|---|---|
Neurological manifestations prior to enrollment | ||
Ataxia | 227 | 162 (71.4) |
Vertical supranuclear gaze palsy | 227 | 160 (70.5) |
Dysarthria | 227 | 156 (68.7) |
Cognitive impairment | 227 | 136 (59.9) |
Dysphagia | 227 | 111 (48.9) |
Dystonia | 227 | 97 (42.7) |
Seizures | 227 | 70 (30.8) |
Clumsiness | 227 | 50 (22.0) |
Cataplexy | 227 | 48 (21.1) |
Behavioral disturbance | 227 | 33 (14.5) |
Psychiatric manifestation | 227 | 26 (11.5) |
Visceral manifestations during infancy | ||
Splenomegaly | 200 | 109 (54.5) |
Hepatomegaly | 203 | 73 (36.0) |
General examination abnormalities at enrollment | ||
Neurological, any | 113 | 92 (81.4) |
Behavioral | 113 | 50 (44.2) |
Musculoskeletal | 190 | 46 (24.2) |
Psychiatric | 191 | 46 (24.1) |
Gastrointestinal | 194 | 25 (12.9) |
Sleep, any | 107 | 17 (15.9) |
Respiratory tract | 197 | 17 (8.6) |
Genitourinary | 182 | 13 (7.1) |
Head/neck | 186 | 11 (5.9) |
Skin | 185 | 9 (4.9) |
Cardiovascular | 198 | 5 (2.5) |
Lymphatic | 175 | 1 (0.6) |