Fig. 4: Clinical trials of two patients with EIMFS with valeriasen.
a, Dosing schedule. b–d, Trends in seizure frequency and duration in patient 1 (b) and patient 2 (c, d) as reported in seizure diaries recorded by the parents. Actual dosing is indicated by red bars. The total number of focal tonic seizures per day was counted. In patient 1, we started to count myoclonic seizures (subtle body movement lasting less than 1 s) from day 241. e–g, Daily seizure counts and binned averages for every 50-day period before and after dosing in patient 1 (e) and patient 2 (f, g). In patient 2, dosing was resumed on day 832. Actual dosing is indicated by red bars. Seizure frequency was significantly reduced after valeriasen treatment (****P < 0.0001; D −44 to 0 versus D1–50 P = 0.0109 in f; D783–832 versus D883–932 P = 0.0370 and D783–832 versus D983–1,032 P = 0.0002 in g). n = 50 in e. D −44 to 0 n = 38, D1–50 n = 33, D51–100 n = 39 and others n = 50 in f. D1,033–1,082 n = 16, D1,133–1,182 n = 43, D1,183–1,232 n = 46, D1,383–1,432 and D1,433–1,482 n = 48, D1,483–1,536 n = 54 and others n = 50 in g. Data are presented as mean values. In e–g, *P < 0.05, ***P < 0.001 and ****P < 0.0001; all significance is compared with the leftmost column in each figure (one-way ANOVA with Dunnett’s multiple comparisons test). Detailed seizure trends by seizure semiology and seizure duration are described in Extended Data Fig. 7a. The seizure type in patient 2 is focal tonic seizures with or without secondary generalized clonic seizures.
