14800-DvRappard

81 Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy 5 Patient ID MLD type Follow-up (months) GMF at diagnosis GMF latest assessment* Cognition at diagnosis (age in years) MRI at diagnosis MRI at latest assessment * Deceased Non-transplanted patients MLD-8 Late- infantile 39 6 6 NA 4 NA yes (5y) MLD 20 Late-infantile 36 6 6 NA 10 NA yes (6y) MLD-26 Late-infantile 72 5 6 NA 17 NA yes (8y) MLD-34 Late-infantile 36 6 6 NA 12 NA yes (4y) MLD-40 Late-infantile 19 6 6 NA 8 NA no MLD-57 Late-infantile 29 4b 6 NA 13 NA no MLD-5 Juvenile 42 1 6 71 (7y) 22 26 no MLD-6 Juvenile 42 1 6 65 (9y) 21 23 no MLD-10 Juvenile 84 1 5 NA 18 NA no MLD-11 Juvenile 84 1 5 NA 14 NA no MLD-12 Juvenile 93 1 6 68 (6y) 12 NA no MLD-17 Juvenile 72 1 6 66 (6y) 14 NA yes (12y) MLD-18 Juvenile 48 1 6 75 (6y) 14 NA yes (12y) MLD-22 Juvenile 44 1 6 65 (15y) 22 NA no MLD-29 Juvenile 22 1 6 61 (7y) 18 NA yes (8y) MLD-33 Juvenile 68 NA 6 NA 15 25 yes (11y) MLD-38 Juvenile 29 1 5 NA 17 NA no MLD-39 Juvenile 33 1 4b 63 (7y) 15 NA no MLD-54 Juvenile 87 1 5 NA 17 19 no MLD-25 Adult 103 1 3 50 (22y) 19 23 no MLD-32 Adult 70 0 0 NA 16 16 no MLD-56 Adult 38 1 2 53 (32y) 13 17 no Abbreviations: GMF, gross motor function; MRI, magnetic resonance imaging; DQ, developmental quotient; NA, not available. * GMF and MRI post HCT are scored at latest follow-up assessment.