Drug class
Duchenne muscular dystrophy exon-skipping antisense oligonucleotides represent a therapeutic class of synthetic, splice-modulating nucleic acid analogues designed to treat specific genetic subtypes of Duchenne muscular dystrophy. Read more about Duchenne Muscular Dystrophy-Exon Skipping Antisense Oligonuc
5 drugs in this class 2 manufacturers
Duchenne muscular dystrophy exon-skipping antisense oligonucleotides represent a therapeutic class of synthetic, splice-modulating nucleic acid analogues designed to treat specific genetic subtypes of Duchenne muscular dystrophy.
These agents—including phosphorodiamidate morpholino oligomers such as eteplirsen, golodirsen, viltolarsen, and casimersen—bind selectively to targeted pre-mRNA sequences within the dystrophin gene.
By sterically hindering the splicing machinery, they induce the exclusion of a specific mutated or flanking exon during transcription, thereby restoring the open reading frame and enabling the production of an internally truncated, partially functional dystrophin protein.
This targeted molecular approach is exclusively indicated to slow disease progression in individuals with Duchenne muscular dystrophy confirmed to have mutations amenable to specific exon-skipping strategies.
Duchenne muscular dystrophy exon-skipping antisense oligonucleotides represent a therapeutic class of synthetic, splice-modulating nucleic acid analogues designed to treat specific genetic subtypes of Duchenne muscular dystrophy These agents—including phosphorodiamidate morpholino oligomers such as eteplirsen, golodirsen, viltolarsen, and casimersen—bind selectively to targeted pre-mRNA sequences within the dystrophin gene By sterically hindering the splicing machinery, they induce the exclusion...
Duchenne muscular dystrophy exon-skipping antisense oligonucleotides represent a therapeutic class of synthetic, splice-modulating nucleic acid analogues designed to treat specific genetic subtypes of Duchenne muscular dystrophy These agents—including phosphorodiamidate morpholino oligomers such as eteplirsen, golodirsen, viltolarsen, and casimersen—bind selectively to targeted pre-mRNA sequences within the dystrophin gene This targeted molecular approach is exclusively indicated to slow disease...
By sterically hindering the splicing machinery, they induce the exclusion of a specific mutated or flanking exon during transcription, thereby restoring the open reading frame and enabling the production of an internally truncated, partially functional dystrophin protein
This targeted molecular approach is exclusively indicated to slow disease progression in individuals with Duchenne muscular dystrophy confirmed to have mutations amenable to specific exon-skipping strategies
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