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Translarna, ataluren (formerly PTC124)

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Company PTC Therapeutics Inc.
DescriptionSmall molecule that facilitates complete translation of proteins containing nonsense mutations
Molecular Target Not available
Mechanism of ActionTranslational bypass therapy
Therapeutic ModalitySmall molecule

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Disease CategoryStandard IndicationIndication DetailsPartnersMilestonesLatest Stage of DevelopmentRegulatory Designation
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