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Company BioMarin Pharmaceutical Inc.
DescriptionSelective histone deacetylase 3 (HDAC3) inhibitor
Molecular Target Histone deacetylase 3 (HDAC3)
Mechanism of ActionHistone deacetylase 3 (HDAC3) inhibitor
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase I
Standard IndicationAtaxia
Indication DetailsTreat Friedreich's ataxia (FRDA)
Regulatory Designation U.S. - Orphan Drug (Treat Friedreich's ataxia (FRDA))

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