Proposed Therapies for Pantothenate-Kinase-Associated Neurodegeneration
2019 ◽
Vol 13
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pp. 117906951985111
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Keyword(s):
Multiple approaches to therapy have been proposed for the rare inherited neurodegenerative disease associated with mutations in the PANK2 gene, called pantothenate-kinase-associated neurodegeneration (PKAN). Penetration of the blood-brain barrier for treatment of a central nervous system (CNS) disorder is a major challenge in drug discovery. Evaluation of the biochemistry and medicinal chemistry of the proposed therapies reveals potential liabilities among several compounds under consideration for clinical development.
2005 ◽
Vol 5
(1)
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pp. 65-72
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1991 ◽
Vol 16
(3)
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pp. 171-175
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2002 ◽
Vol 304
(3)
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pp. 1268-1274
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2016 ◽
Vol 240
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pp. 251-266
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Keyword(s):
2018 ◽
Vol 283
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pp. 126-134
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1998 ◽
Vol 13
(6)
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pp. 287-293
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Keyword(s):