Last Posted: Sep 23, 2024
- Wilson and Jungner Revisited: Are Screening Criteria Fit for the 21st Century?
- Advancing ASO therapies from development to implementation
- Rare developmental disorder caused by variants in a small RNA gene
- FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
- Health Care Costs After Genome-Wide Sequencing for Children With Rare Diseases in England and Canada
- Equity in action: The Diagnostic Working Group of The Undiagnosed Diseases Network International
- Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review.
Qin Xiang Ng et al. Health Econ Rev 2024 14(1) 42 - Exploring the benefits, harms and costs of genomic newborn screening for rare diseases.
Emma L Baple et al. Nat Med 2024 - Global health for rare diseases through primary care
- The landscape for rare diseases in 2024
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