Casimersen (AMONDYS 45™): An Antisense Oligonucleotide for Duchenne Muscular Dystrophy

Casimersen (AMONDYS 45™): An Antisense Oligonucleotide for Duchenne Muscular Dystrophy

Casimersen (AMONDYS 45 ) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass developed by Sarepta therapeutics. It was approved by the Food and Drug Administration (FDA) in February 2021 to treat Duchenne muscular dystrophy (DMD) in patients whose gene mutation is...

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Bibliographic Details
Published in:Biomedicines Vol. 12; no. 4; p. 912
Main Authors: Assefa, Milyard, Gepfert, Addison, Zaheer, Meesam, Hum, Julia M, Skinner, Brian W
Format: Journal Article
Language:English
Published: Switzerland MDPI AG 01-04-2024
Subjects:
Age
AMONDYS 45
antisense oligonucleotide
Antisense oligonucleotides
Antisense therapy
casimersen
Clinical trials
CRISPR
Duchenne muscular dystrophy
Duchenne's muscular dystrophy
Dystrophin
exon skipping
FDA approval
Females
Fomivirsen
Immune system
Males
Mipomersen
mRNA
Muscular dystrophy
Mutation
Myocytes
Pathophysiology
Pegaptanib
Point mutation
Proteins
Steroids
X chromosomes
phosphorodiamidate oligomer
antisense oligonucleotide
exon skipping
Duchenne muscular dystrophy
neuromuscular disorders
casimersen
dystrophin
AMONDYS 45
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Description
Summary:Casimersen (AMONDYS 45 ) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass developed by Sarepta therapeutics. It was approved by the Food and Drug Administration (FDA) in February 2021 to treat Duchenne muscular dystrophy (DMD) in patients whose gene mutation is amenable to exon 45 skipping. Administered intravenously, casimersen binds to the pre-mRNA of the gene to skip a mutated region of an exon, thereby producing an internally truncated yet functional dystrophin protein in DMD patients. This is essential in maintaining the structure of a myocyte membrane. While casimersen is currently continuing in phase III of clinical trials in various countries, it was granted approval by the FDA under the accelerated approval program due to its observed increase in dystrophin production. This article discusses the pathophysiology of DMD, summarizes available treatments thus far, and provides a full drug review of casimersen (AMONDYS 45 ).
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ISSN:2227-9059
2227-9059
DOI:10.3390/biomedicines12040912