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Orphanet Journal of Rare Diseases|September 12, 2023
Financing repurposed drugs for rare diseases: a case study of Unravel BiosciencesBechara Abouarab, Christian Bazarian, Zied Ben Chaouch, et al.Nature Biotechnology|December 9, 2017
Just how good an investment is the biopharmaceutical sector?Richard T Thakor, Nicholas Anaya, Yuwei Zhang, et al.Plos One|December 28, 2020
A cost/benefit analysis of clinical trial designs for COVID-19 vaccine candidatesDonald A Berry, Scott Berry, Peter Hale, et al.Cell|September 4, 2012
Single-stranded siRNAs activate RNAi in animalsWalt F Lima, Thazha P Prakash, Heather M Murray, et al.Nucleic Acids Research|January 26, 2020
Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotidesMichael E Østergaard, Cheryl L De Hoyos, W Brad Wan, et al.Nature Medicine|August 9, 2024
Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorderAlban Ziegler, Joanne Carroll, Jennifer M Bain, et al.Circulation Research|April 2, 2013
Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humansMark J Graham, Richard G Lee, Thomas A Bell, et al.Nucleic Acids Research|February 5, 2021
Site-specific incorporation of 5'-methyl DNA enhances the therapeutic profile of gapmer ASOsGuillermo Vasquez, Graeme C Freestone, W Brad Wan, et al.Lancet (London, England)|March 16, 2010
Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trialFrederick J Raal, Raul D Santos, Dirk J Blom, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 1, 2016
Integrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human VolunteersStanley T Crooke, Brenda F Baker, T Jesse Kwoh, et al.Pageof 17