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Company BioMarin Pharmaceutical Inc.
DescriptionRNA-based antisense oligonucleotide
Molecular Target Huntingtin mRNA ; Huntingtin (HTT)
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPreclinical
Standard IndicationHuntington's disease (HD)
Indication DetailsTreat Huntington's disease (HD)
Regulatory Designation

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