ABSTRACT Background X‐linked adrenoleukodystrophy (X‐ALD) is a neurometabolic disorder caused by pathogenic variants in ABCD1, leading to slowly progressive spinal cord disease in nearly all affected men. Sensitive biomarkers to quantify disease severity and predict progression are needed for clinical care and trial design.
Eda G. Kabak +4 more
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Jiang Y, Wang T, Chen X, Liang J.
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Xia F, Mari T, Gunasagaran S.
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Stoor-Burning E +3 more
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Xiong Y, Sirintrapun SJ.
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