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Published in 2020 at "Journal of Inherited Metabolic Disease"
DOI: 10.1002/jimd.12226
Abstract: All men and most women with X‐linked adrenoleukodystrophy (ALD) develop myelopathy in adulthood. As clinical trials with new potential disease‐modifying therapies are emerging, sensitive outcome measures for quantifying myelopathy are needed. This prospective cohort study…
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Keywords:
cord;
severity myelopathy;
measure;
csa ... See more keywords