The long-awaited solution for personalized medicine
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Published in | Frontiers in science (Lausanne) Vol. 3 |
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Main Authors | , |
Format | Journal Article |
Language | English |
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Frontiers Media S.A
26.06.2025
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Online Access | Get full text |
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Author | Khorkova, Olga Wahlestedt, Claes |
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Cites_doi | 10.1016/S2214-109X(24)00056-1 10.57264/cer-2023-0187 10.1007/s40273-022-01197-9 10.1056/NEJMoa1813279 10.1186/s13023-017-0695-0 10.1038/s41573-023-00704-7 10.1007/s12325-023-02621-y 10.3389/fsci.2025.1458636 10.1016/j.ajhg.2025.02.017 |
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References_xml | – volume: 12 start-page: e341 year: 2024 ident: B6 article-title: The landscape for rare diseases in 2024 publication-title: Lancet Glob Health doi: 10.1016/S2214-109X(24)00056-1 – volume: 13 start-page: e230187 year: 2024 ident: B7 article-title: Evaluation of inpatient and emergency department healthcare resource utilization and costs pre- and post-nusinersen for the treatment of spinal muscular atrophy using United States claims publication-title: J Comp Eff Res doi: 10.57264/cer-2023-0187 – volume: 41 year: 2023 ident: B9 article-title: Caregiver burden of spinal muscular atrophy: a systematic review publication-title: Pharmacoeconomics doi: 10.1007/s40273-022-01197-9 – volume: 381 year: 2019 ident: B4 article-title: Patient-customized oligonucleotide therapy for a rare genetic disease publication-title: N Engl J Med doi: 10.1056/NEJMoa1813279 – volume: 12 start-page: 141 year: 2017 ident: B10 article-title: Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain publication-title: Orphanet J Rare Dis doi: 10.1186/s13023-017-0695-0 – volume: 22 year: 2023 ident: B3 article-title: Amplifying gene expression with RNA-targeted therapeutics publication-title: Nat Rev Drug Discov doi: 10.1038/s41573-023-00704-7 – volume-title: Electronic Code of Federal Regulations. National Archives year: 2025 ident: B5 article-title: Part 211—Current good manufacturing practice for finished pharmaceuticals [21 CFR Part 211] – volume: 40 year: 2023 ident: B8 article-title: Health care resource utilization and costs for patients with spinal muscular atrophy: findings from a retrospective US claims database analysis publication-title: Adv Ther doi: 10.1007/s12325-023-02621-y – volume: 3 year: 2025 ident: B1 article-title: NANOSPRESSO: toward personalized, locally produced nucleic acid nanomedicines publication-title: Front Sci doi: 10.3389/fsci.2025.1458636 – volume: 112 year: 2025 ident: B2 article-title: Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments publication-title: Am J Hum Genet doi: 10.1016/j.ajhg.2025.02.017 |
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Title | The long-awaited solution for personalized medicine |
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