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Rational Design of Novel Therapies for Pantothenate Kinase–Associated Neurodegeneration

This review highlights the recent scientific advances that have enabled rational design of novel clinical trials for pantothenate kinase‐associated neurodegeneration (PKAN), a rare autosomal recessive neurogenetic disorder associated with progressive neurodegenerative changes and functional impairment. PKAN is caused by genetic variants in the PANK2 gene that result in dysfunction in pantothenate kinase 2 (PANK2) enzyme activity, with consequent disruption of coenzyme A (CoA) synthesis, and subsequent accumulation of brain iron. The clinical phenotype is varied and may include dystonia, rigidity, bradykinesia, postural instability, spasticity, loss of ambulation and ability to communicate, feeding difficulties, psychiatric issues, and cognitive and visual impairment. There are several symptom‐targeted treatments, but these do not provide sustained benefit as the disorder progresses.