Newest therapeutic options for adrenoleukodystrophy
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X-linked adrenoleukodystrophy is a genetic disease correlated with mutation of ATP-binding cassette, which results in errors of peroxisomal beta oxidation and accumulation of impaired very long chain fatty acids. This leads to degeneration of adrenal glands, spinal cord and myelin sheaths. Despite nearly 100 years of ALD history, the treatment is limited to few therapeutic options, mainly Lorenzo’s Oil and Hematopoetic stem cell transplant. Available therapy can only slow the progression of the disease in early stages. The aim of our study was to present the newest therapeutic options in X-ALD. Substantial articles on new treatment of X-ALD from period 2007-2018 in the Asian, European and American regions have been analyzed. Among 219 articles in PubMed Medline database related to therapy and treatment of X-ALD, 13 articles were selected for analysis, reviewed and divided into two main groups: cause-related treatment (11 articles) and symptoms-related treatment (2 articles). Within cause-related treatment, the usage of known medications such as eg. pioglitazone, natural phenols - resveratrol, gene therapy with adenoassociated virus serotype 9 or combined therapies (Hematopoetic Stem Cell Gene Therapy) have been reviewed. Symptoms related treatment attempted to reduce spasticity and secondary dystonia in X-ALD patients. Reviewed research presents progress in development of treatment options for X-ALD, however still in primary in vitro and animal tested stages. Secondary neurological symptoms medication is awaiting for better solutions for ALD patients, in order to improve their Quality of Life and allow symptomless course for a longer time.
- I Faculty of Medicine, Medical University of Lublin, Al. Raclawickie 1, 20-059 Lublin, Poland
- Pediatric Neurology Department, III Chair of Pediatrics, Medical University of Lublin, 6 Prof. A. Gebali 6 Str., 20-093 Lublin, Poland
- Department of Pediatric Endocrinology and Diabetology with Endocrine - Metabolic Laboratory, III Chair of Pediatrics, Medical University of Lublin, 6 Prof. A. Gebali 6 Str., 20-093 Lublin, Poland
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