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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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2019
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Acesso em linha: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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