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Company BioBlast Pharma Ltd.
DescriptionMitochondrial fusion protein comprised of a trans-activator of transcription peptide, a heterologous mitochondrial targeting sequence peptide and a functional frataxin (FXN; FRDA) protein
Molecular Target
Mechanism of Action 
Therapeutic ModalityBiologic: Fusion protein
Latest Stage of DevelopmentPreclinical
Standard IndicationAtaxia
Indication DetailsTreat Friedreich's ataxia
Regulatory Designation

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