PMC9830009_01
PMC9830009_02
Gross motor function measurement at baseline (prior to elamipretide dose) and during elamipretide treatment (Case 2)
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After 1 year and 3 months of therapy at this dose, the patient has demonstrated developmental progress and a lack of continued regression (Table 2 and Figure 1).
PMC9830009_03
Severity of illness score during the first 4 months of treatment with elamipretide (Case 3)
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Clinical Global Impression scale with the severity of illness (global) score improving from "markedly ill" to "borderline ill" during treatment (Figure 2). Cardiac improvements included an increase in the LV end-diastolic internal dimension (Z-score from -2.7 to +0.7), a decrease ventricular septal thickness (Z-score from 4.7 to 3.5), and a decrease in LV posterior wall thickness (Z-score from 5.8 to 4.7).