Pantothenate kinase activation restores brain coenzyme A in a mouse model of pantothenate kinase associated neurodegeneration
Article in Journal of Pharmacology and Experimental Therapeutics (October 2023)
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Study design challenges and strategies in clinical trials for rare diseases: Lessons learned from pantothenate kinase-associated neurodegeneration
Article in Frontiers in Neurology (March 2023)
PKAN pathogenesis and treatment
Article in Molecular Genetics and Metabolism (October 2022)