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Readable research linked to original sources
Browse normalized, publication-ready research with direct links to its evidence and source.
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Readable research linked to original sources
Browse normalized, publication-ready research with direct links to its evidence and source.
2 articles
Newest firstBackgroundNucleic acid therapies (NATs) comprise engineered DNA- or RNA-based medicines that act through sequence-specific interactions to modify gene function. Among these, antisense oligonucleotide (ASO) therapies are designed to bind messenger RNA (mRNA) or pre-mRNA to alter splicing, transcript stability, or translation. Many patients with a rare genetic disease stand to benefit from these treatments and, as u…
Open article record in new tab ↗IntroductionGovernments create policies to address societal needs and then assess their effectiveness through policy metrics. The UK Rare Diseases Framework and its England Rare Diseases Action Plans aim to improve the lives of people with lived experience of rare diseases and their carers. Reaching these goals requires more effective engagement with those least likely to interact with NHS services. This paper out…