2023
DOI: 10.21203/rs.3.rs-2536822/v1
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Treatment of Pantothenate-kinase neurodegeneration with baclofen, botulinum toxin, and deferiprone: A case report

Abstract: Pantothenate kinase-associated neurodegeneration (PKAN) is a rare autosomal recessive disorder characterized by progressive motor symptoms such as dystonia and spasticity. Classical PKAN is the most common subtype of neurodegeneration with brain iron accumulation (NBIA). There is no current established treatment for PKAN. Baclofen and botulinum toxin have been reported to improve motor symptoms and ease care in these patients. Deferiprone is a well-tolerated iron chelator and has been shown effective in reduci… Show more

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