X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...

X-Linked Adrenoleukodystrophy (X-ALD ... A public health program that tests newborns for certain genetic, endocrine, and metabolic disorders shortly after birth to enable early diagnosis and ...

X-ALD is a CNS demyelinating disorder ... “This is the first time that a severe disease of the brain has been treated with success by gene therapy,” says Aubourg. He points out, however ...

ACSL4 = acyl-CoA synthetase long-chain; ALD = alcoholic liver disease; CPT1A = carnitine palmitoyl transferase 1A; CTGF = connective tissue growth factor; DDX5 = DEAD box polypeptide 5 ...

After their son’s diagnosis, the Hall family is raising millions for ALD research—giving kids a fighting chance at life.

ALD is an X-linked metabolic disease affecting 1 in 21,000 newborn boys worldwide, of whom around 40% go on to developed cALD. In patient with the disease a genetic mutation causes the build-up of ...

It’s a rare genetic disorder that impacts a small population, and due to limited research, much remains unknown about ...

Inspired by their 9-month old son William, who was diagnosed with ALD after a newborn screening, the Hall family seeks to jumpstart research into the rare disease, in partnership with The Dallas ...

For several decades, the prevalence of steatotic liver disease (SLD) has been steadily increasing.1 Alterations in hepatic ...

X-linked adrenoleukodystrophy is a genetic disorder that mainly affects ... VLCFAs are considered biomarkers of disease in patients with X-ALD. Treatment with VK0214 resulted in significant ...