Orphanet Journal of Rare Diseases

Table 1 Current therapeutic options

From: Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

	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

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ISSN: 1750-1172

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