Table 1.

Current therapeutic options

	Late-infantile			Early-juvenile			Late-juvenile			Adult
Disease stage	Pre-	Early	Late	Pre-	Early	Late	Pre-	Early	Late	Pre-	Early	Late
Supportive care	✔	✔	✔	✔	✔	✔	✔	✔	✔	✔	✔	✔
HSCT	?	✖	✖	✔	✔	✖	✔	✔	✖	✔	✔	✖
Ex vivo GT	✔	✖	✖	✔	✔	✖	Trial	Trial	✖	?	?	✖
ERT	?	Trial	Trial	?	?	?	?	?	?	?	?	?

Trial Currently investigated

✔ Eligible

✖ Not eligible

? Not investigated or debatable indication