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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Dades bibliogràfiques
Publicat a:Orphanet J Rare Dis
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2019
Matèries:
Research
Accés en línia: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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