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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Detaylı Bibliyografya
Yayımlandı:Orphanet J Rare Dis
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2019
Konular:
Research
Online Erişim: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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