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Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.

Daphne H SchoenmakersFanny MochelLaura A AdangJaap-Jan BoelensValeria CalbiErik A EklundSabine W GrønborgFrancesca FumagalliSamuel GroeschelCaroline LindemansCaroline SevinLudger SchölsDipak RamAyelet ZeremHolm GraessnerNicole I Wolf
Published in: Orphanet journal of rare diseases (2024)
HSCT for MLD is available in most European countries, but uncertainties exist in Eastern and South-Eastern Europe. Applied eligibility criteria and management vary and may not align with the latest scientific insights, indicating physicians' struggle in providing evidence-based care. Interaction between local physicians and international experts is crucial for adequate treatment decision-making and cross-border care in the rapidly changing MLD field.
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