QR kód

Systemic administration of the antisense oligonucleotide NS‐089/NCNP‐02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial

Abstract Aim The purpose of this study is to evaluate the safety and pharmacokinetics of the novel morpholino oligomer NS‐089/NCNP‐02 which can induce exon 44 skipping, in patients with DMD. Additionally, we aimed to identify markers predictive of therapeutic efficacy and determine the optimal dosin...

Celý popis

Uloženo v:
Hlavní autoři: Takami Ishizuka, Hirofumi Komaki, Yasuko Asahina, Harumasa Nakamura, Norio Motohashi, Eri Takeshita, Yuko Shimizu‐Motohashi, Akihiko Ishiyama, Chihiro Yonee, Shinsuke Maruyama, Eisuke Hida, Yoshitsugu Aoki
Médium: Artigo
Jazyk:Inglês
Vydáno: Wiley 2023-06-01
Edice:Neuropsychopharmacology Reports
Témata:
antisense oligonucleotide
DMD
Duchenne muscular dystrophy
exon 44
exon‐skipping
first‐in‐human study
On-line přístup:https://doi.org/10.1002/npr2.12335
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo vytvoří štítek k tomuto záznamu!

Podobné jednotky