From roadmap to a sustainable end-to-end individualized therapy pathway.
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| Title: | From roadmap to a sustainable end-to-end individualized therapy pathway. |
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| Authors: | Jonker AH; Health Technologies and Services Department, Techmed Centre, University of Twente, Enschede, The Netherlands., Tataru EA; Fondation Maladies Rares, Paris, France., Dimmock DP; Creyon Bio Inc, San Diego, CA, USA., Bateman-House A; Division of Medical Ethics, Department of Population Health, Grossman School of Medicine, NYU Langone Health, New York, NY, USA., Graessner H; Centre for Rare Diseases, University Hospital Tübingen, Tübingen, Germany., Baynam G; Rare Care Centre, Perth Children's Hospital, Perth, Australia., Augustine EF; Kennedy Krieger Institute, Baltimore, MD, USA., Jaffe A; School of Clinical Medicine, Faculty of Medicine and Health, University of New South Wales, Sydney, NSW, Australia., Pasmooij AMG; Dutch Medicines Evaluation Board, Utrecht, The Netherlands.; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, The Netherlands., Iliach O; Certara, Toronto, ON, Canada., Horgan R; Cure Rare Diseases, Woodbridge, CT, USA., Davies J; MRC Molecular Haematology Unit, University of Oxford, Oxford, UK., Mitkus S; Global Genes, Washington, DC, USA., Lapteva L; US Food and Drug Administration, Silver Spring, MD, USA., Synofzik M; Division of Translational Genomics of Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany.; German Center for Neurodegenerative Diseases (DZNE), Tübingen, Germany., Yu TW; Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., O'Connor D; The Association of British Pharmaceutical Industry, London, UK., Aartsma-Rus A; Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZH Leiden, The Netherlands. |
| Source: | Therapeutic advances in rare disease [Ther Adv Rare Dis] 2025 May 27; Vol. 6, pp. 26330040251339204. Date of Electronic Publication: 2025 May 27 (Print Publication: 2025). |
| Publication Type: | Journal Article; Review |
| Journal Info: | Publisher: Sage Country of Publication: England NLM ID: 9918557474706676 Publication Model: eCollection Cited Medium: Internet ISSN: 2633-0040 (Electronic) Linking ISSN: 26330040 NLM ISO Abbreviation: Ther Adv Rare Dis Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2633-0040 |
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| DOI: | 10.1177/26330040251339204 |
