A call to arms against ultra-rare diseases
- 4 June 2021
- journal article
- editorial
- Published by Springer Science and Business Media LLC in Nature Biotechnology
- Vol. 39 (6), 671-677
- https://doi.org/10.1038/s41587-021-00945-0
Abstract
No abstract availableKeywords
This publication has 19 references indexed in Scilit:
- A Case for n-of-1 Trials—ReplyJAMA Internal Medicine, 2019
- Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer ExperienceNucleic Acid Therapeutics, 2019
- Rare Diseases: Addressing the Challenges in Diagnosis, Drug Approval, and Patient AccessValue in Health, 2018
- The Effects of 2′-O-Methoxyethyl Oligonucleotides on Renal Function in HumansNucleic Acid Therapeutics, 2018
- The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical TrialsNucleic Acid Therapeutics, 2017
- Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human VolunteersMolecular Therapy, 2016
- The Cockayne Syndrome Natural History (CoSyNH) study: clinical findings in 102 individuals and recommendations for careGenetics in Medicine, 2016
- Rare Disease Terminology and Definitions—A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest GroupValue in Health, 2015
- Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness researchJournal of Clinical Epidemiology, 2013
- The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?Personalized Medicine, 2011