Disease progression in women with X-linked adrenoleukodystrophy is slow

Abstract Background Over 80% of women with X-linked adrenoleukodystrophy (ALD) develop spinal cord disease in adulthood for which treatment is supportive only. For future clinical trials quantitative data on disease progression rates are essential. Moreover, diagnosis can be challenging in ALD women...

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Bibliographic Details
Main Authors: Irene C. Huffnagel, Marcel G. W. Dijkgraaf, Georges E. Janssens, Michel van Weeghel, Björn M. van Geel, Bwee Tien Poll-The, Stephan Kemp, Marc Engelen
Format: Article
Published: BMC 2019-02-01
Series:Orphanet Journal of Rare Diseases
Online Access:http://link.springer.com/article/10.1186/s13023-019-1008-6