NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
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Related Access Program
Related Clinical Trial
Testing of NBIA Genes: Analysis of Genetic Heterogeneity and Validation of Mitochondrial Markers for Assessing Causality of Sequence Variants.
NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
Imaging Neuromelanin and Iron in Dystonia/Parkinsonism
CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
Compassionate Use of Deferiprone in Patients With PKAN
Brain Perfusion in Pantothenate Kinase-associated Neurodegeneration (PKAN)
Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients