Proposed Therapies for Pantothenate-Kinase-Associated Neurodegeneration
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) disor...
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| Format: | Article |
| Language: | English |
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SAGE Publishing
2019-05-01
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| Series: | Journal of Experimental Neuroscience |
| Online Access: | https://doi.org/10.1177/1179069519851118 |
| Summary: | 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. |
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| ISSN: | 1179-0695 |