DMD, dystrophin, 1756

N. diseases: 484; N. variants: 345
Disease: Hemophilia, NOS ×
Source: ALL
Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
CUI: C0684275
Disease: Hemophilia, NOS
Hemophilia, NOS 		0.010 AlteredExpression disease BEFREE While minimally impacting the normal transcriptome, Upf3b-ASO treatment significantly stabilizes the PTC-containing dystrophin mRNA in mdx mice and coagulation factor IX mRNA in a hemophilia mouse model. 29334995 2018