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RE-024

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Collapse Summary General Information
Company Retrophin Inc.
DescriptionPhosphopantothenate molecule that restores disruption in coenzyme A biosynthesis caused by a mutant pantothenate kinase 2 (PANK2; NBIA1) gene
Molecular Target Pantothenate kinase 2 (PANK2) (NBIA1)
Mechanism of Action 
Therapeutic Modality 
Latest Stage of DevelopmentPreclinical
Standard Indication Neurology (unspecified)
Indication DetailsTreat pantothenate kinase-associated neurodegeneration (PKAN)
Regulatory Designation

Partner


 Product Details
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