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Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON)
Date du début: 1 nov. 2011, Date de fin: 31 oct. 2015,
Neurodegeneration with brain iron accumulation (NBIA) is a heterogeneous group of rare hereditary neurodegenerative disorders characterized by high levels of brain iron. The most common form is pantothenate kinase-associated neurodegeneration (PKAN). Classic PKAN and most other NBIA cases are characterised by early childhood onset and rapid progression. Currently, there is no proven therapy to hal ...
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