Search Results - "rare diseases"

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  1. 301
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    Current genetic diagnostics in inborn errors of immunity by Sandra von Hardenberg, Isabel Klefenz, Doris Steinemann, Nataliya Di Donato, Ulrich Baumann, Bernd Auber, Christian Klemann, Christian Klemann

    Published 2024
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  2. 302
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    Orphan Drugs, Compounded Medication and Pharmaceutical Commons by Kim Hendrickx, Marc Dooms

    Published 2021
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  3. 303
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    Clusters of rare disorders and congenital anomalies in South America by Augusto César Cardoso-, Guillermo Reales, Lavinia Schuler-Faccini

    Published 2023
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  4. 304
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    Improving translational studies: lessons from rare neuromuscular diseases by Dominic Wells

    Published 2015
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  5. 305
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    Orphan drug policy analysis in China by Meilin Liu, Yanqin Lu, Yanqin Lu, Junfeng Li, Yongtao Zhang, Shanshan Zhang, Yisheng Liu

    Published 2024
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  6. 306
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    Increasing differential diagnosis between lipoma and liposarcoma through radiomics: a narrative review by Raffaele Natella, Giulia Varriano, Maria Chiara Brunese, Marcello Zappia, Michela Bruno, Michele Gallo, Flavio Fazioli, Igino Simonetti, Vincenza Granata, Luca Brunese, Antonella Santone

    Published 2023
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  7. 307
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    Health economic benefits through the use of diagnostic support systems and expert knowledge by Tina Willmen, Lukas Völkel, Simon Ronicke, Martin C. Hirsch, Jessica Kaufeld, Reinhard P. Rychlik, Annette D. Wagner

    Published 2021
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  8. 308
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    Mucopolysaccharidosis IIIB and mild skeletal anomalies: coexistence of NAGLU and CYP26B1 missense variations in the same patient in a Chinese family by Jinliang Li, Han Xie, Yuwu Jiang

    Published 2018
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  9. 309
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    Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs-a Systematic Literature Review. Next Steps in Methodology Development? by Aleksandra Baran-Kooiker, Marcin Czech, Marcin Czech, Coen Kooiker

    Published 2018
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  10. 310
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    Work participation in adults with rare genetic diseases - a scoping review by Gry Velvin, Brede Dammann, Trond Haagensen, Heidi Johansen, Hilde Strømme, Amy Østertun Geirdal, Trine Bathen

    Published 2023
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  11. 311
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    Drug Repurposing Using Molecular Network Analysis Identifies Jak as Targetable Driver in Necrobiosis Lipoidica by Alysia N. Hughes, Xing Li, Julia S. Lehman, Steven A. Nelson, David J. DiCaudo, Rekha Mudappathi, Angelina Hwang, Jacob Kechter, Mark R. Pittelkow, Aaron R. Mangold, Aleksandar Sekulic

    Published 2024
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  12. 312
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    Doenças raras: o que o pediatra necessita saber by Maria Goretti Moreira Guimarães Penido, Maria Helena Vaisbich, Lilian Monteiro Pereira Palma, Nilzete Liberato Bresolin

    Published 2024
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  13. 313
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    The German government's global health strategy - a strategy also to support research and development for neglected diseases? by Angela Fehr, Oliver Razum

    Published 2014
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  14. 314
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    Health technology assessment (HTA) and economic evaluation: efficiency or fairness first by Jeff Richardson, Michael Schlander

    Published 2019
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  15. 315
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    Importance of national and international registries of inborn errors of metabolism by Roberto Giugliani

    Published 2014
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  16. 316
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    Comparative Analysis for the Performance of Variant Calling Pipelines on Detecting the de novo Mutations in Humans by Yu Liang, Li He, Yiru Zhao, Yinyi Hao, Yifan Zhou, Menglong Li, Chuan Li, Xuemei Pu, Zhining Wen

    Published 2019
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  17. 317
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    Navigating the unknown: how to best 'reflect' standard of care in indications without a dedicated treatment pathway in health technology assessment submissions by Grammati Sarri, Maria Rizzo, Sheela Upadhyaya, Victoria Federico Paly, Luis Hernandez

    Published 2024
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  18. 318
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    REASONS FOR DIFFICULTY OF THE PHARMACOECONOMIC ANALYSIS FOR ORPHAN DRUGS. WAYS FOR SOLUTIONS by Andrey Vladislavovich Pavlysh, Aleksei Sergeevich Kolbin, Roman Andreevich Gapeshin, Stanislav Mikhailovich Malyshev

    Published 2015
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  19. 319
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    Editorial: New Generation Therapeutic Modalities by Peter Timmins

    Published 2018
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  20. 320
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    Current Problems in Provision of Orphan Drugs and Ways to Solve Them by Alexei S. Kolbin, Roman A. Gapeshin, Stanislav M. Malyshev

    Published 2016
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