Advertisement

The clinical course of childhood and adolescent adrenoleukodystrophy before and after Lorenzo's oil

      Abstract

      The clinical course of Japanese patients with childhood and adolescent adrenoleukodystrophy (ALD) who were treated with Lorenzo's oil after the progression of early symptoms was investigated by Kaplan–Meier analyses. Many of the early symptoms, including visual, personality, hearing, mental and speech disturbances, had already appeared when the oil therapy was started. These early symptoms appeared significantly faster than bed-ridden state or dysphagia (P<0.01). Gait disturbance appeared faster than bed-ridden state and dysphagia (P<0.05), and tended to appear later than the early symptoms. These results would reflect the early natural course of childhood and adolescent ALD before treatment, and provide a basis for the evaluation of therapeutic trials for ALD.

      Keywords

      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Brain and Development
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Moser H.W
        • Smith K.D
        • Moser A.B
        X-linked adrenoleukodystrophy.
        in: Scriver C.R Beaudet A.L Sly W.S Valle D The metabolic and molecular bases of inherited disease. 7th ed. McGraw–Hill, New York1995: 2325-2349
        • Mosser J
        • Douar A.M
        • Sarde C.O
        • Kioschis P
        • Feil R
        • Moser H
        • et al.
        Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters.
        Nature. 1993; 361: 726-730
        • Braun A
        • Ambach H
        • Kammerer S
        • Rolinski B
        • Stockler S
        • Rabl W
        • et al.
        Mutations in the gene for X-linked adrenoleukodystrophy in patients with different clinical phenotypes.
        Am J Hum Genet. 1995; 56: 854-861
        • Rizzo W.B
        • Leshner R.T
        • Odone A
        • Dammann A.L
        • Craft D.A
        • Jensen M.E
        • et al.
        Dietary erucic acid therapy for X-linked adrenoleukodystrophy.
        Neurology. 1989; 39: 1415-1422
        • Suzuki Y
        • Shimozawa N
        • Yajima S
        • Inoue K
        • Orii T
        • Kondo N
        Incidence of peroxisomal disorders in Japan.
        Jpn J Hum Genet. 1996; 41: 167-175
        • Bezman L
        • Moser H.W
        Incidence of X-linked adrenoleukodystrophy and the relative frequency of its phenotypes.
        Am J Med Genet. 1998; 76: 415-419
        • Aubourg P
        • Blanche S
        • Jambaque I
        • Rocchiccioli F
        • Kalifa G
        • Naud-Saudreau C
        • et al.
        Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation.
        N Engl J Med. 1990; 322: 1860-1866
        • Suzuki Y
        • Isogai K
        • Teramoto T
        • Tashita H
        • Shimozawa N
        • Nishimura M
        • et al.
        Bone marrow transplantation for the treatment of X-linked adrenoleukodystrophy.
        J Inherit Metab Dis. 2000; 23: 453-458
        • Singh I
        • Khan M
        • Key L
        • Pai S
        Lovastatin for X-linked adrenoleukodystrophy.
        N Engl J Med. 1998; 339: 702-703
        • Kemp S
        • Wei H.M
        • Lu J.F
        • Braiterman L.T
        • McGuinness M.C
        • Moser A.B
        • et al.
        Gene redundancy and pharmacological gene therapy: implications for X-linked adrenoleukodystrophy.
        Nat Med. 1998; 4: 1261-1268