X-linked adrenoleukodystrophy

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Related to adrenoleukodystrophy: Lorenzo's Oil

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.
Endocrine Dysfunctions in Patients with Inherited Metabolic Diseases
* The report provides a snapshot of the global therapeutic landscape of Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline Review, H1 2016
Schaumburg, "The testis in adrenoleukodystrophy," American Journal of Pathology, vol.
Testosterone-induced effects on lipids and inflammation
In a new report, doctors detail the cases of two boys from France with adrenoleukodystrophy who underwent gene therapy.
Gene therapy helps two kids suffering from fatal brain disease
Adrenoleukodystrophy (ALD) is a rare, inherited metabolic disorder.
'Nothing could be done by the time Lawrence was diagnosed' The Oscar-nominated film Lorenzo's Oil told the true story of a couple's search for a cure for their son's rare genetic condition. But for those young boys already showing symptoms of ALD, the oil offers little hope of a cure. Health Editor Madeleine Brindley spoke to Virginia Prifti about the reality of the disease
Together with her husband Augusto, Mrs Odone developed a way to alleviate the rare genetic disorder adrenoleukodystrophy which struck their son Lorenzo in 1984.
Lorenzo film mother dies
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.
Dishy Doctor George may return to ER
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.
Magenta Therapeutics Presents New Data from Phase 2 Study of MGTA-456 Cell Therapy in Patients with Inherited Metabolic Disorders
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.
FDA grants Breakthrough Therapy Designation for bluebird bio's Lenti-D
(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)?
Newborn Screening for Lysosomal Storage Disorders: Quo Vadis?
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.
Applied Genetic Technologies swings to net income of USD3.0m for the three months ended 31 December 2015
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