COMPOSITIONS USEFUL FOR MODULATING SPLICING

The present application discloses compounds of Formula (I) 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, such as Spinocerebellar Ataxia 3 (SCA3 or Machado-Joseph Diseas...

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Bibliographische Detailangaben
Hauptverfasser:	RATNI, Hasane, KONETZKI, Ingo, LUCAS, Brian, LUZZIO, Michael
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	CHEMISTRY HETEROCYCLIC COMPOUNDS HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY ORGANIC CHEMISTRY PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Beschreibung
Zusammenfassung:	The present application discloses compounds of Formula (I) 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, such as Spinocerebellar Ataxia 3 (SCA3 or Machado-Joseph Disease). La présente invention concerne des composés de formule (I) qui modulent l'épissage d'un pré-ARNm, codé par des gènes, et des méthodes de traitement de maladies et d'affections associées à l'expression génique ou à l'activité de protéines codées par des gènes, telles que l'ataxie spinocérébelleuse 3 (ASC3 ou maladie de Machado-Joseph).