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

When considering genetic modifiers of ALD and NAFLD, one should first consider those genes that influence etiologic factors causally linked to the disease. These are exemplified by the associated ...

Aedrik Quinn, 8, has the rare life-threatening disease cerebral adrenoleukodystrophy (ALD). This is the first of a series of ...

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 ...

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 ...

The result of the NEXUS trial means that the two companies can now press on with their plan to refile for EU approval of the ...

The FDA last week announced that it is looking into the safety of bluebird bio ’s gene therapy Skysona (elivaldogene ...

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 ...

The prevalence of and mortality from alcohol-associated liver disease (ALD) and alcohol use disorder (AUD) increased among older U.S. adults, according to a study presented at The Liver Meeting ...

Chronic liver disease (CLD ... particularly in MASH and ALD. The review highlights the importance of understanding this gene's function to unravel new therapeutic targets. The study was recently ...

ALD and MetALD affect much lower ... found a much lower rate of Hispanics, 29%, had the disease at that time. The Virginia researchers say genetics, as well as relatively high rates of obesity ...