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

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

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
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	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
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	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
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	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
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	MC: No change

ISSN: 2053-8871