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...
|Main Authors:||, , , , , , ,|
|Series:||Orphanet Journal of Rare Diseases|