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Subcutaneous PRO053

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Company BioMarin Pharmaceutical Inc.
DescriptionAntisense oligoribonucleotide that skips exon 53 on the dystrophin gene
Molecular Target
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPhase I/II
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation
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