The X-ALD gene encodes a peroxisomal membrane protein (ALDP) that is part of a small family of related peroxisomal membrane proteins. We show that 4-phenylbutyrate treatment of cells from both X ...

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 is a genetic disorder that mainly affects the nervous system and the adrenal glands, which are located on top of each kidney. Also Read: Viking Therapeutics Stock ...

Michael Conway paid tribute as he revealed a special connection with the late Pope Francis which gave him hope to fight ...

Gene therapies have already been tested for other forms of ALD, which include cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease, as well as adrenal ...

Kyrie Vincent is 9 years old, and has been diagnosed with Adrenoleukodystrophy (ALD), which is a rare, genetic disorder that affects the nervous system and adrenal glands. ALD is the result of a ...

The brain disease X-linked ALD can be treated with a combination of gene therapy and blood stem cell therapy, new EU-funded research shows.

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.