Therapy of X‐linked adrenoleukodystrophy: Prognosis based upon age and MRI abnormality and plans for placebo‐controlled trials

Evaluation of the therapy of X‐linked adrenoleukodystrophy (X‐ALD) is hampered by its rarity and by the striking and unpredictable variation in phenotypic expression. We present two approaches that may facilitate therapy evaluation. (1) We have analysed data on 377 X‐ALD patients who have been follo...

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Veröffentlicht in:	Journal of inherited metabolic disease 2000-05, Vol.23 (3), p.273-277
Hauptverfasser:	Moser, H. W., Bezman, L., Lu, S. E., Raymond, G. V.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adrenoleukodystrophy - pathology Adrenoleukodystrophy - physiopathology Age Factors Biological and medical sciences Clinical Trials as Topic Degenerative and inherited degenerative diseases of the nervous system. Leukodystrophies. Prion diseases Humans Magnetic Resonance Imaging Medical sciences Multicenter Studies as Topic Neurology Prognosis X Chromosome
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