The not-for profit foundation n-Lorem seeks to provide experimental antisense oligonucleotide treatments to patients with ultra-rare disease free for their entire lifetime.
This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Enriched phenotypes in rare variant carriers suggest pathogenic mechanisms in rare disease patients
BioData Mining Open Access 17 January 2025
-
Invisible patients in rare diseases: parental experiences with the healthcare and social services for children with rare diseases. A mixed method study
Scientific Reports Open Access 18 June 2024
-
Towards ethical drug pricing: the European Orphan Genomic Therapies Fund
Gene Therapy Open Access 24 April 2024
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$32.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on SpringerLink
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Nguengang Wakap, S. et al. Eur. J. Hum. Genet. 28, 165–173 (2020).
Jo, A. et al. BMJ Open 9, e027248 (2019).
Copley-Merriman, K. Value Health 21, 491–492 (2018).
Richter, T. et al. Value Health 18, 906–914 (2015).
Crooke, S. T., Seth, P. P., Vickers, T. A. & Liang, X. H. J. Am. Chem. Soc. 142, 14754–14771 (2020).
Crooke, S. T., Liang, X. H., Crooke, R. M., Baker, B. F. & Geary, R. S. Biochem. Pharmacol. 2020, 114196 (2020).
Crooke, S.T., Baker, B.F., Crooke, R.M. & Liang, X.H. Nature Rev. Drug Discov. https://doi.org/10.1038/s41573-021-00162-z (2021).
Crooke, S.T., Liang, X.H., Baker, B.F. & Crooke, R.M. J. Biol. Chem. https://doi.org/10.1016/j.jbc.2021.100416 (2021).
Crooke, S. T., Vickers, T. A. & Liang, X. H. Nucleic Acids Res. 48, 5235–5253 (2020).
Margolis, A. & Giuliano, C. Epilepsy Behav. Rep. 12, 100336 (2019).
Kravitz, R. L., Sim, I. & Duan, N. JAMA Intern. Med. 179, 453–453 (2019).
Lillie, E. O. et al. Per. Med. 8, 161–173 (2011).
Duan, N., Kravitz, R. L. & Schmid, C. H. J. Clin. Epidemiol. 66 (Suppl.), S21–S28 (2013).
Crooke, S. T. et al. Mol. Ther. 24, 1771–1782 (2016).
Crooke, S. T. et al. Nucleic Acid Ther. 27, 121–129 (2017).
Crooke, S. T. et al. Nucleic Acid Ther. 28, 10–22 (2018).
Crooke, S. T. et al. Nucleic Acid Ther. 29, 16–32 (2019).
Wilson, B. T. et al. Genet. Med. 18, 483–493 (2016).
Vessoni, A. T., Guerra, C. C. C., Kajitani, G. S., Nascimento, L. L. S. & Garcia, C. C. M. Genet. Mol. Biol. 43 (Suppl. 1), e20190085 (2020).
Acknowledgements
I thank our donors, collaborators, volunteers, board of directors and Access To Treatment Committee members. Thanks also to Kim Butler for excellent assistance and Rosanne Crooke for editorial comment.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
S.T.C. is executive chairman of the Board of Directors at Ionis Pharmacueticals, Inc.
Rights and permissions
About this article
Cite this article
Crooke, S.T. A call to arms against ultra-rare diseases. Nat Biotechnol 39, 671–677 (2021). https://doi.org/10.1038/s41587-021-00945-0
Published:
Issue date:
DOI: https://doi.org/10.1038/s41587-021-00945-0
This article is cited by
-
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap
Nature Reviews Drug Discovery (2025)
-
Enriched phenotypes in rare variant carriers suggest pathogenic mechanisms in rare disease patients
BioData Mining (2025)
-
Invisible patients in rare diseases: parental experiences with the healthcare and social services for children with rare diseases. A mixed method study
Scientific Reports (2024)
-
Enhancing pediatric access to cell and gene therapies
Nature Medicine (2024)
-
Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder
Nature Medicine (2024)
