Original paper
Natural history of a cohort of ABCD 1 variant female carriers
Abstract
Background and purpose The therapeutic scenario of X‐linked adrenoleukodystrophy (X‐ ALD ) is rapidly changing. Whereas the disease is well characterized in men, the condition remains to be fully clarified in women carrying ATP binding cassette subfamily D member 1 ( ABCD1 ) variants. Specifically, data on clinical progression are needed, in order to recommend any appropriate management. The objective of this study was to outline the natural...
Paper Details
Title
Natural history of a cohort of ABCD 1 variant female carriers
Published Date
Nov 9, 2018
Volume
26
Issue
2
Pages
326 - 332
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Notes
History