Table 2. Clinical parameters.

		1	2	3	4	5	6	7
Cardiac Status - LVMI g/m2	Baseline	160	446	277	410	317	347	220
	Current (week)	81	80	65	92	164	108	83
Respiratory Status	Baseline	O2	O2 and BiPAP at night	O2	Invasively ventilated	Invasively ventilated	Invasively ventilated	No support required
	Current	No respiratory support	O2 and BiPAP at night	No respiratory support	Off ventilator 10–12 hours a day	O2 and BiPAP at night	BiPAP at night	Invasive ventilation at week 38 ERT until death 2 months later
Motor Status	Baseline	Head lag and severe hypotonia and motor delay	Head lag, antigravity movements arms>legs,	Severe hypotonia, floppy baby, no head or neck control	Axial hypotonia, withdraws extremities to stimulation, contractures of large joint of upper and lower extremities, weak grasp	Head lag unable to sit or roll over	Severe hypotonia, Antigravity movement in arms	Unable to independently hold head or sit unsupported
	Current	Minor head lag when pulled to sit, rolls over, lifts head from prone, sits unsupported, cruises, briefly stands unsupported	Bears weight independently	Ambulates independently	Sits with support; minimal capacity for weight bearing on lower extremities	Standing with support	Marked axial and peripheral hypotonia, yet able to move arms against gravity; near-complete lower extremity immobility	Not able to independently hold head or sit unsupported
Feeding Status	Baseline	NG tube feeds at age 2 months for dysphagia and fatigue.	NG tube feeds due to aspiration	NG tube feeds started at age 1.4 months	Baseline – G-tube feeds and continues	NJ feeds started at age 3 months	Aspiration and penetration with feeding and started on NG tube feeds at baseline.	NG tube feeds at age 3 months due to aspiration
	Current	Oral feeds	GJ tube	Oral feeds	G tube	G tube	G-tube feeds	GJ tube