Disease progression in women with X-linked adrenoleukodystrophy is slow

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, as the...

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書目詳細資料
發表在:Orphanet J Rare Dis
Main Authors: Huffnagel, Irene C., Dijkgraaf, Marcel G. W., Janssens, Georges E., van Weeghel, Michel, van Geel, Björn M., Poll-The, Bwee Tien, Kemp, Stephan, Engelen, Marc
格式: Artigo
語言:Inglês
出版: BioMed Central 2019
主題:
Research
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6367840/
https://ncbi.nlm.nih.gov/pubmed/30732635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-019-1008-6
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