Table 4 Patient-reported outcomes for a patient with HPDL encephalopathy treated with 4-HB

From: Coenzyme Q headgroup intermediates can ameliorate a mitochondrial encephalopathy

Patient-reported outcomes

Day 2

Day 12

Day 30

Day 54

Day 124

Day 250

Stance with one foot forward (time (s) for right, left)

57, 60

190, 190

*

*

NA

NA

Tandem stance (time (s) for right, left)

Unable

21, 30

*

*

NA

NA

Falls

Daily

Daily

Weekly

Weekly

Rare

Rare

Able to run

No

No

No

Yes

Yes

Yes

Able to ride a bicycle

No

No

No

No

Yes

Yes

Able to step laterally to catch a ball

No

No

No

No

No

Yes

  1. *Not quantified as the patient became bored and started walking after standing for 3 min (one foot forward) or 30 s (tandem stance). NA, not applicable.