454 results for Rare+Diseases+-+drug+therapy

454 results for Rare+Diseases+-+drug+therapy

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      ArticlesPeer ReviewedFull Text

      Author / Creator:Dharssi, Safiyya ; Wong-Rieger, Durhane ; Harold, Matthew ; Terry, Sharon
      Publisher:England: BioMed Central
      Journal title:Orphanet journal of rare diseases, 2017-03, Vol.12 (1), p.63-63, Article 63
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      Author / Creator:DeFrancesco, Laura
      Publisher:United States: Nature Publishing Group
      Journal title:Nature biotechnology, 2022-11, Vol.40 (11), p.1539-1541
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      Author / Creator:Cully, Megan
      Publisher:London: Nature Publishing Group UK
      Journal title:Nature reviews. Drug discovery, 2019-10, Vol.18 (11), p.811-813
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      Author / Creator:Levine, James A. ; Stemitsiotis, Clea
      Publisher:London: Nature Publishing Group UK
      Journal title:Nature reviews. Drug discovery, 2024-01, Vol.23 (1), p.10-11
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      Author / Creator:Kranz, Philip ; McGauran, Natalie ; Banzi, Rita ; Ünal, Can ; Lotz, Fabian ; Kaiser, Thomas
      Publisher:England: British Medical Journal Publishing Group
      Journal title:BMJ (Online), 2023-05, Vol.381, p.e072796-e072796
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      Author / Creator:Michaeli, Thomas ; Jürges, Hendrik ; Michaeli, Daniel Tobias
      Publisher:England: British Medical Journal Publishing Group
      Journal title:BMJ (Online), 2023-05, Vol.381, p.e073242
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      Author / Creator:O’Connor, Daniel J. ; Gabaldo, Michela ; Aartsma-Rus, Annemieke ; Hechtelt Jonker, Anneliene
      Publisher:London: Nature Publishing Group UK
      Journal title:Nature reviews. Drug discovery, 2023-11, Vol.22 (11), p.857-858
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      Author / Creator:Fermaglich, Lewis J ; Miller, Kathleen L
      Publisher:England: BioMed Central Ltd
      Journal title:Orphanet journal of rare diseases, 2023-06, Vol.18 (1), p.163-163, Article 163
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      Author / Creator:Chen, Rui ; Liu, Sen ; Han, Jiashu ; Zhou, Shuhua ; Liu, Yang ; Chen, Xiaoyuan ; Zhang, Shuyang
      Publisher:London: Nature Publishing Group UK
      Journal title:Nature reviews. Drug discovery, 2024-03, Vol.23 (3), p.168-169
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      Author / Creator:Hechtelt Jonker, Anneliene ; Hivert, Virginie ; Gabaldo, Michela ; Batista, Liliana ; O’Connor, Daniel ; Aartsma-Rus, Annemieke ; Day, Simon ; Sakushima, Ken ; Ardigo, Diego
      Publisher:London: Nature Publishing Group UK
      Journal title:Nature reviews. Drug discovery, 2020-08, Vol.19 (8), p.495-496
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      Author / Creator:Belot, Alexandre ; Benezech, Sarah ; Tusseau, Maud
      Publisher:England: Elsevier Ltd
      Journal title:The Lancet (British edition), 2024-02, Vol.403 (10427), p.592-593
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      Author / Creator:Costa, Enrico ; Moja, Lorenzo ; Wirtz, Veronika J ; van den Ham, Hendrika A ; Huttner, Benedikt ; Magrini, Nicola ; Leufkens, Hubert Gm
      Publisher:Switzerland: World Health Organization
      Journal title:Bulletin of the World Health Organization, 2024-01, Vol.102 (1), p.22-31
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      Author / Creator:Hillmann, Petra ; Fabbro, Doriano
      Publisher:Switzerland: MDPI AG
      Journal title:International journal of molecular sciences, 2019-11, Vol.20 (22), p.5792
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      Author / Creator:Miller, Kathleen L ; Lanthier, Michael
      Publisher:United States: The People to People Health Foundation, Inc., Project HOPE
      Journal title:Health affairs (Millwood, Va.), 2024-01, Vol.43 (1), p.18-5
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      Author / Creator:Hughes-Wilson, Wills ; Palma, Ana ; Schuurman, Ad ; Simoens, Steven
      Publisher:England: BioMed Central
      Journal title:Orphanet journal of rare diseases, 2012-09, Vol.7 (1), p.74-74
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      Author / Creator:Yoneyama, Koichiro ; Schmitt, Christophe ; Kotani, Naoki ; Levy, Gallia G. ; Kasai, Ryu ; Iida, Satofumi ; Shima, Midori ; Kawanishi, Takehiko
      Publisher:Cham: Springer International Publishing
      Journal title:Clinical pharmacokinetics, 2018-09, Vol.57 (9), p.1123-1134
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      Author / Creator:Jayasundara, Kavisha ; Hollis, Aidan ; Krahn, Murray ; Mamdani, Muhammad ; Hoch, Jeffrey S ; Grootendorst, Paul
      Publisher:England: BioMed Central Ltd
      Journal title:Orphanet journal of rare diseases, 2019-01, Vol.14 (1), p.12-12, Article 12
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      Author / Creator:Hendriksz, Christian J. ; Burton, Barbara ; Fleming, Thomas R. ; Harmatz, Paul ; Hughes, Derralynn ; Jones, Simon A. ; Lin, Shuan-Pei ; Mengel, Eugen ; Scarpa, Maurizio ; Valayannopoulos, Vassili ; Giugliani, Roberto ; Slasor, Peter ; Lounsbury, Debra ; Dummer, Wolfgang
      Publisher:Dordrecht: Springer Netherlands
      Journal title:Journal of inherited metabolic disease, 2014-11, Vol.37 (6), p.979-990
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      Author / Creator:Wagner, Michael J ; Othus, Megan ; Patel, Sandip P ; Ryan, Chris ; Sangal, Ashish ; Powers, Benjamin ; Budd, G Thomas ; Victor, Adrienne I ; Hsueh, Chung-Tsen ; Chugh, Rashmi ; Nair, Suresh ; Leu, Kirsten M ; Agulnik, Mark ; Sharon, Elad ; Mayerson, Edward ; Plets, Melissa ; Blanke, Charles ; Streicher, Howard ; Chae, Young Kwang ; Kurzrock, Razelle
      Publisher:England: BMJ Publishing Group Ltd
      Journal title:Journal for immunotherapy of cancer, 2021-08, Vol.9 (8), p.e002990
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      Author / Creator:Brasil, Sandra ; Pascoal, Carlota ; Francisco, Rita ; Dos Reis Ferreira, Vanessa ; Videira, Paula A ; Valadão, And Gonçalo
      Publisher:Switzerland: MDPI AG
      Journal title:Genes, 2019-11, Vol.10 (12), p.978
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