Articles tagged with Adrenoleukodystrophy
A Kennedy Krieger researcher's work on a promising oral drug, leriglitazone, has shown it can slow the progression of cerebral adrenoleukodystrophy (cALD) in all 11 trial participants. Nearly half of boys treated with the medication appeared to halt disease progression entirely.
A new model of chromosome X-linked adrenoleukodystrophy in C. elegans has been identified, accelerating research on the disease and its potential pharmacological targets. The model points to glial cells as responsible for neurological damage, with oxidative stress caused by mitochondria being a major cause.
Researchers investigated the role of mitochondria in adrenoleukodystrophy, a disease causing spinal cord degeneration. They found that a diabetes drug, pioglitazone, halted nerve fibre degeneration by preventing mitochondrial loss and oxidative stress.
IDIBELL has licensed a patent for a repositioned drug to treat X-linked adrenoleukodystrophy, a rare neurodegenerative disease. The patented product has shown promise in laboratory studies, but further research is needed before its application can be considered.
Gene therapy has been successfully tested in France, halting the progression of adrenoleukodystrophy in two children. The innovative approach uses a modified HIV virus to deliver a therapeutic gene to bone marrow cells, which then migrate to the brain and display correcting potential.
Researchers have achieved significant success with gene therapy in treating adrenoleukodystrophy (ALD), a potentially fatal brain disorder. The treatment has arrested the progression of the disease in two boys, showing no adverse effects to date.