Table 2 Schedule of events

Informed consent

X

Demographic information

X

Medical and surgical history

X

Inclusion exclusion

X

Vital signs

X

X

X

X

X

Physical exam

X^c

X^c

X

X

X

Pregnancy test^d

X

X

X

X

10 Meter walk

X

100 m timed

X

X

X

X

X

NSAD

X

X

X

X

X

4 stair climb

X

X

X

X

X

Timed Up and Go (TUG)

X

X

X

X

X

Handheld dynamometry

X

X

X

X

X

Performance Upper Limb PUL 2.0

X

X

X

X

X

9 hole peg test

X

X

X

X

X

Spirometry

X

X

X

X

X

Concomitant meds

X

X

X

X

X

Domain delta questionnaire

X

X

X

X

Patient reported outcome measures

X^c

X^c

X

X

X

Urine biomarkers

X

X

X

Blood biomarkers

X

X

X

X

Prior muscle biopsy acquired (if performed)

X

Review adverse events

X

X

X

X