Casimersen (Amondys 45™) is an antisense oligonucleotide of the phosphorodiamidate
morpholino oligomer subclass developed by Sarepta Therapeutics for the treatment of …

Antisense oligonucleotides (ASOs) are used to selectively inhibit the translation of disease-
associated genes via Ribonuclease H (RNaseH)-mediated cleavage or steric hindrance …

Abstract Viltolarsen (Viltepso® in Japan) is a phosphorodiamidate morpholino antisense
oligonucleotide being developed by Nippon Shinyaku, in collaboration with the National …

Lipid-based nanoparticles (LBNPs) are biocompatible and biodegradable vesicles that are
considered to be one of the most efficient drug delivery platforms. Due to the prominent …

Dystrophic epidermolysis bullosa (DEB) is one of the major types of EB, a rare hereditary
group of trauma-induced blistering skin disorders. DEB is caused by inherited pathogenic …

Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …

Epidermolysis bullosa (EB) is a heterogeneous group of rare inherited blistering skin
disorders characterized by skin fragility following minor trauma, usually present since birth …

2019 has been an excellent year in terms of peptides and oligonucleotides (TIDES)
approved by the FDA. Despite the drop in the number of total drugs approved by the FDA in …

Epidermolysis bullosa (EB) is a group of rare genetic disorders for which significant progress
has been achieved in the development of molecular therapies in the last few decades. Such …

Background Joubert syndrome and related disorders are a group of ciliopathies
characterized by mid-hindbrain malformation, developmental delay, hypotonia, oculomotor …