Addressing the Needs of Patients with Ultra-Rare Mutations One Patient at a Time: The n-Lorem Approach
Author:
Affiliation:
1. n-Lorem Foundation, Carlsbad, California, USA.
Publisher
Mary Ann Liebert Inc
Subject
Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry
Link
https://www.liebertpub.com/doi/pdf/10.1089/nat.2021.0046
Reference21 articles.
1. A call to arms against ultra-rare diseases
2. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database
3. Prevalence and practice for rare diseases in primary care: a national cross-sectional study in the USA
4. Rare Diseases: Addressing the Challenges in Diagnosis, Drug Approval, and Patient Access
5. Rare Disease Terminology and Definitions—A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group
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