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X chromosome–linked adrenoleukodystrophy (ADL) is a devastating neurologic disorder with an estimated birth incidence of 1 in 17,000 newborns. It is a metabolic disorder that impairs peroxisomal beta-oxidation of very-long-chain fatty acids. These accumulate in plasma and all tissues, including the white matter of the brain, spinal cord, and adrenal cortex. ADL is caused by mutation in the ABCD1 gene located on the X chromosome. Clinically, patients have a cognitive deficit that includes compromise of visuomotor and visuospatial functions, attention, and reasoning.
In this manufacturer-sponsored study, 17 patients aged 4 to 13 years with cerebral ADL were enrolled to receive the investigational Lenti-D gene therapy, which involved infusion o…