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 ...

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 ...

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 ...

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

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 ...

The 77 boys were treated for cerebral adrenoleukodystrophy (CALD), a formerly incurable and progressive genetic brain disease ...

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 ...