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Nature Medicine|July 21, 2026
Individualized antisense oligonucleotides for SCN2A-related developmental epileptic encephalopathyOlivia Kim-McManus, Laurence Mignon, Julie Douville, et al.
Drug Discovery Today|June 16, 2026
Estimating the global inventory of deprioritized clinical-stage drug development programs: Toward a market for shelved assetsAnnette Bakker, Tanisha Carino, Joonhyuk Cho, et al.
Lancet (London, England)|September 26, 2016
Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trialsNicholas J Viney, Julian C van Capelleveen, Richard S Geary, et al.
Nucleic Acids Research|April 30, 2019
Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteinsMichael T Migawa, Wen Shen, W Brad Wan, et al.
Therapeutic Innovation & Regulatory Science|August 27, 2022
Leveraging Patient Preference Information in Medical Device Clinical Trial DesignLiliana Rincon-Gonzalez, Wendy K D Selig, Brett Hauber, et al.
Nature Biotechnology|May 1, 2019
Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic indexWen Shen, Cheryl L De Hoyos, Michael T Migawa, et al.
Nucleic Acids Research|November 11, 2015
Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcriptsSebastien A Burel, Christopher E Hart, Patrick Cauntay, et al.
Nucleic Acids Research|August 21, 2021
Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotidesBrooke A Anderson, Graeme C Freestone, Audrey Low, et al.
Patterns (New York, N.Y.)|August 25, 2021
Predicting drug approvals: The Novartis data science and artificial intelligence challengeKien Wei Siah, Nicholas W Kelley, Steffen Ballerstedt, et al.
Plos One|July 2, 2025
Financing drug development via adaptive platform trialsJoonhyuk Cho, Eugene Sorets, Shomesh Chaudhuri, et al.
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