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NDC Symposium

Leukodystrophies

Leukodystrophies amass a heterogeneous group of over 50 genetic disorders of myelination that lead to progressive degeneration of the white matter in the central nervous system and often also involve the peripheral nervous system. Currently, the therapeutic window for most leukodystrophies is brief and may simply involve the management of symptoms of palliative care. Up and coming approaches to treat these brain disorders include gene replacement, stem cell therapy, gene editing, and high throughput screening-based individualized medicine approaches; some of which are now in clinical trials. Leukodystrophies take an enormous toll on affected patients and their families, making expert diagnosis and management essential. The combined incidence of leukodystrophies is relatively high, such that most child neurologists are likely to encounter leukodystropy cases reasonably often. Nonetheless, the high number of individual diseases and genetic mutations can make it difficult to properly diagnose these conditions in a timely manner. Given that most leukodystrophies have early onset phenotypes, child neurologists are the likely specialists to diagnose and provide care for these patients. Including leukodystrophies in the NDC symposia will give child neurologists the opportunity to align on the latest evidence and research in this field. Moreover, conversations about leukodystrophy research, both in the clinic and in the laboratory, will enhance our understanding of normal brain development and oligodendroglial cell biology. It will have been 15 years since NDC focused on the topic of leukodystrophies and advances over those 15 years will be central to our discussions in Kansas City. For additional resources on leukodystrophies, please consult the World Leukodystrophy Alliance and the Leukodystrophy Care Network/Hunter's Hope websites.



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