Significant gains in our understanding of the biochemical defects in PKAN have shifted the therapeutic focus from palliative care to rational interventions. Novel therapies aimed at correcting specific perturbations in cellular metabolism hold great promise and are currently under investigation. Treatment of this rare neurode-generative disorder is likely to change significantly over the next decade.
|Original language||English (US)|
|Title of host publication||Treatment of Pediatric Neurologic Disorders|
|Number of pages||5|
|ISBN (Print)||0824726936, 9780824726935|
|State||Published - Jan 1 2005|
ASJC Scopus subject areas