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Table 1 Demographic and clinical characteristics of this series of FRDA patients

From: IGF-1 in Friedreich’s Ataxia – proof-of-concept trial

Patients Age Gender Disease duration Initial SARA GAA repeats ECG Initial LVMI (g/m 2) IGF-1 therapy (dose) Follow-up Therapy withdrew Adverse events SF36v2:
PC: Physical component
MC: Mental component
Patient 1 23 F 12 18 >800 Widespread inferolateral T-wave inversion Normal 50 μg/kg bid 3 four-month period Four-month period 3 None PC: feeling fair
MC: feeling fair
Patient 2 24 F 14 21.5 >500 Widespread inferolateral T-wave inversion Normal 50 μg/kg bid 3 four-month period Four-month period 3 None PC: feeling better
MC: feeling better
Patient 3 26 M 4 13 >500 Widespread inferolateral T-wave inversion Normal 50 μg/kg bid 3 four-month period Four-month period 3 GH-secreting pituitary adenoma PC: No change
MC: No change
Patient 4 36 F 14 14.25 >500 Normal Normal 50 μg/kg bid 3 four-month period Ongoing None No change during therapy period, later worsening
Patient 5 24 F 3 9 >500 Widespread inferolateral T-wave inversion Normal 50 μg/kg bid 3 four-month period Four-month period 3 None PC: No change
MC: No change