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Sep 2026

Allogeneic haematopoietic stem cell transplantation in late-onset metachromatic leukodystrophy.

Comparing the long-term outcomes of the largest European cohort of HSCT-treated and untreated late-juvenile and adult MLD patients provides an evidence base for future comparison with innovative therapies and underscores the importance of early diagnosis and intervention.

D. Schoenmakers, M. Datema, Shanice Beerepoot et al. · 0 citations

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