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

Viking Therapeutics has announced positive results from its Phase Ib clinical trial of VK0214, a thyroid hormone receptor ...

X-Linked Adrenoleukodystrophy (ALD) is a rare genetic disorder that breaks down myelin, the protective sheath that surrounds the brain and spinal cord. Early intervention and regular screenings ...

Allogeneic hematopoietic stem cell transplantation (HCT) has been shown to have disease-modifying effects in the demyelinating cerebral form of X-linked adrenoleukodystrophy (X-ALD), but finding a ...

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