Table 5.

Components of Pillar 3, Treatment.

Parameter	Description	Score	Interaction
Availability	An EMA-approved treatment is available	1.5	AND
	A treatment intervention is available (diet, HSCT, BMT)	1
	A treatment is in late development (phase 3)	1
	A treatment is in early development (preclinical, phase 1, or phase 2)	0.5
Outcomes	The treatment strategy changes the prognosis for all forms of the disorder	1.5	OR
	The treatment strategy changes the prognosis for some forms of the disorder	1
	The treatment strategy does not change prognosis or improves only some symptoms of the disorder	0.5
	Pre-symptomatic initiation results in better outcomes	1	AND