X-linked adrenoleukodystrophy

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X-linked adrenoleukodystrophy

[¦eks ‚liŋkt ə‚drē·nō‚lü·kə′dis·trə·fē]
(medicine)
An inherited peroxisomal disorder in which a defective peroxisomal membrane protein results in impaired ?-oxidation of very long chain fatty acids, which subsequently accumulate in the adrenal glands, nervous system, and testes and disrupt normal activity.
McGraw-Hill Dictionary of Scientific & Technical Terms, 6E, Copyright © 2003 by The McGraw-Hill Companies, Inc.
References in periodicals archive ?
Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison's disease improves survival and neurological outcomes.
* The report provides a snapshot of the global therapeutic landscape of Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)
Schaumburg, "The testis in adrenoleukodystrophy," American Journal of Pathology, vol.
In a new report, doctors detail the cases of two boys from France with adrenoleukodystrophy who underwent gene therapy.
Together with her husband Augusto, Mrs Odone developed a way to alleviate the rare genetic disorder adrenoleukodystrophy which struck their son Lorenzo in 1984.
The drama begins when an eight-year-old boy is rushed into the emergency room of Chicago's County General Hospital one night with ALD - adrenoleukodystrophy - from which sufferers die an agonising death.
The ongoing Phase 2 study in inherited metabolic disorders aims to enroll 12 patients with cerebral adrenoleukodystrophy, metachromatic leukodystrophy or globoid cell leukodystrophy, and previously also enrolled patients with Hurler's syndrome.
Food and Drug Administration has granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy, a rare, serious and life-threatening hereditary neurological disorder.
(d) Why would you invest in fluorometry when many of the assays now being discussed and emerging in the future can only be carried out using MS (X-linked adrenoleukodystrophy, MPS-IVA, Niemann-Pick type C)?
Biotechnology company Applied Genetic Technologies Corporation (NasdaqGM:AGTC) Monday updated the financial results for the quarter ended 31 December 2015 and also announced that Biogen (NasdaqGS:BIIB) has selected the company's adrenoleukodystrophy (ALD) as the first of the three discovery programmes.