Table 3.

A suggested example of assessments to monitor patients receiving vosoritide and limb surgery in any combination

Assessment	Before treatment		During treatment		After treatment
	Essential	Desirable	Essential	Desirable	Essential	Desirable
Physical examination
Anthropometry (which may include height; growth curve monitoring; sitting height and arm span; weight; body mass index; waist, hip, chest, and head circumference)	x		x		x
Body proportions	x		x		x
Bone assessment (which may include length, AGV)	x		x		x
Functional recovery (including assessment of ADL, personal hygiene, age-related participation in sports and social activities)	N/A			x		x
HRQol variables (incl. PROs)	x		x		x
Radiological assessments
Radiological variables (which may include lower limb alignment, spine parameters)	x		x		x
Annual X-Ray to assess bone formation		x	x		x
Bone age (Dimeglio method, X-Ray of olecranon)	x		x		x
Quality of callus*	N/A		x		x
Surgery-specific assessments
Deformity assessment†	x		x		x
Soft tissue evaluation‡	x		x		x
Joint stability‡	x		x		x
Muscle function¶		x		x		x
Range of motion (may include gait study)	x		x		x
Bone healing*	N/A		x		x
Safety
Safety (including assessment of medical and surgical complications**)	x		x		x
Complementary assessments
Physiotherapy	x		x		x
Blood and urine parameters		x		x		x
Whole spine MRI		x				x

*Assessed by a validated method, such that by as Venkatesh et al.; [60] †May include clinical, radiographic, or gait examination; ‡Based on clinical examination; ¶ Muscle strength is graded according to the Medical Research Council (MRC) scale with zero being no contraction and five representing normal strength⁶¹; **Assessed by Paley [62], or alternative appropriate validated assessment [63]