Adrenal Leukodystrophy and Women

Dr Shoji Tsuji. Tokyo University Hospital. The International University of Health and Welfare Genomic Medicine Research Institute. Aadrenal leukodystrophy originally developed in boys of school age, and is called the white matter  Inflammatory demyelination in the area where nerve fibers are gathered and pass) is a disease in which the nervous system covering the nerve…

Current and Future Pharmacological Treatment Strategies in X-Linked Adrenoleukodystrophy

Abstract DMutations in the ABCD1 gene cause the clinical spectrum of the neurometabolic disorder X-linked adrenoleukodystrophy/adrenomyeloneuropathy (X-ALD/AMN). Currently, the most efficient therapeutic opportunity for patients with the cerebral form of X-ALD is hematopoietic stem cell transplantation and possibly gene therapy of autologous hematopoietic stem cells. Both treatments, however, are only accessible to a subset of…