Votoplam (also known as PTC518) is an investigational oral small molecule drug being developed by PTC Therapeutics for the treatment of Huntington's disease (HD). The compound functions as a huntingtin (HTT) gene modulator and splicing factor modifier, designed to selectively reduce huntingtin mRNA and protein levels.
Votoplam (also known as PTC518) is an investigational oral small molecule drug being developed by PTC Therapeutics for the treatment of Huntington's disease (HD). The compound functions as a huntingtin (HTT) gene modulator and splicing factor modifier, designed to selectively reduce huntingtin mRNA and protein levels.
Source: Wikipedia "Votoplam" · CC BY-SA 4.0
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