CONDENSED PYRIDAZINE AMINE DERIVATIVES TREATING SCA3

Described herein are compounds that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes.

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Bibliographische Detailangaben
Hauptverfasser: RATNI, Hasane, KONETZKI, Ingo, LUCAS, Brian, LUZZIO, Michael
Format: Patent
Sprache:eng ; fre ; ger
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Beschreibung
Zusammenfassung:Described herein are compounds that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes.