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

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

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    Gale Encyclopedia of Science.
    ebooks

    Publisher:FARMINGTON HILLS : Cengage Gale, 2021.
    Edition:6th ed.
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    Author / Creator:Bronstein, Max G. ; Kakkis, Emil D.
    Publisher:London: Nature Publishing Group UK
    Journal title:Nature reviews. Drug discovery, 2016-11, Vol.15 (11), p.731-732
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    Author / Creator:STOLK, Pieter ; WILLEMEN, Marjolein J. C ; LEUFKENS, Hubert G. M
    Publisher:Genève: Organisation mondiale de la santé
    Journal title:Bulletin of the World Health Organization, 2006-09, Vol.84 (9), p.745-751
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    Author / Creator:Dolgin, Elie
    Publisher:New York: Nature Publishing Group US
    Journal title:Nature medicine, 2010-08, Vol.16 (8), p.837-837
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    Author / Creator:Dugger, Sarah A. ; Platt, Adam ; Goldstein, David B.
    Publisher:London: Nature Publishing Group UK
    Journal title:Nature reviews. Drug discovery, 2018-03, Vol.17 (3), p.183-196
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    ArticlesPeer ReviewedFull Text

    Author / Creator:Kim, Jinkuk ; Hu, Chunguang ; Moufawad El Achkar, Christelle ; Black, Lauren E ; Douville, Julie ; Larson, Austin ; Pendergast, Mary K ; Goldkind, Sara F ; Lee, Eunjung A ; Kuniholm, Ashley ; Soucy, Aubrie ; Vaze, Jai ; Belur, Nandkishore R ; Fredriksen, Kristina ; Stojkovska, Iva ; Tsytsykova, Alla ; Armant, Myriam ; DiDonato, Renata L ; Choi, Jaejoon ; Cornelissen, Laura ; Pereira, Luis M ; Augustine, Erika F ; Genetti, Casie A ; Dies, Kira ; Barton, Brenda ; Williams, Lucinda ; Goodlett, Benjamin D ; Riley, Bobbie L ; Pasternak, Amy ; Berry, Emily R ; Pflock, Kelly A ; Chu, Stephen ; Reed, Chantal ; Tyndall, Kimberly ; Agrawal, Pankaj B ; Beggs, Alan H ; Grant, P. Ellen ; Urion, David K ; Snyder, Richard O ; Waisbren, Susan E ; Poduri, Annapurna ; Park, Peter J ; Patterson, Al ; Biffi, Alessandra ; Mazzulli, Joseph R ; Bodamer, Olaf ; Berde, Charles B ; Yu, Timothy W
    Publisher:United States: Massachusetts Medical Society
    Journal title:The New England journal of medicine, 2019-10, Vol.381 (17), p.1644-1652
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    Author / Creator:Tambuyzer, Erik ; Vandendriessche, Benjamin ; Austin, Christopher P. ; Brooks, Philip J. ; Larsson, Kristina ; Miller Needleman, Katherine I. ; Valentine, James ; Davies, Kay ; Groft, Stephen C. ; Preti, Robert ; Oprea, Tudor I. ; Prunotto, Marco
    Publisher:London: Nature Publishing Group UK
    Journal title:Nature reviews. Drug discovery, 2020-02, Vol.19 (2), p.93-111
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    Author / Creator:Luzzatto, Lucio ; Hyry, Hanna I ; Schieppati, Arrigo ; Costa, Enrico ; Simoens, Steven ; Schaefer, Franz ; Roos, Jonathan C P ; Merlini, Giampaolo ; Kääriäinen, Helena ; Garattini, Silvio ; Hollak, Carla E ; Remuzzi, Giuseppe ; Barbui, Tiziano ; Benigni, Ariela ; Costa, Enrico ; Daina, Erica ; Garattini, Silvio ; Gramaglia, Donatella ; Hollak, Carla E M ; Hyry, Hanna ; Kaarinen, Helena ; Licht, Christoph ; Luzzatto, Lucio ; Merlini, Giampaolo ; Notaro, Rosario ; Remuzzi, Giuseppe ; Schaefer, Franz ; Schieppati, Arrigo ; Simoens, Steven
    Publisher:England: Elsevier Ltd
    Journal title:The Lancet (British edition), 2018-09, Vol.392 (10149), p.791-794
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    Author / Creator:Ory, Daniel S ; Ottinger, Elizabeth A ; Farhat, Nicole Yanjanin ; King, Kelly A ; Jiang, Xuntian ; Weissfeld, Lisa ; Berry-Kravis, Elizabeth ; Davidson, Cristin D ; Bianconi, Simona ; Keener, Lee Ann ; Rao, Ravichandran ; Soldatos, Ariane ; Sidhu, Rohini ; Walters, Kimberly A ; Xu, Xin ; Thurm, Audrey ; Solomon, Beth ; Pavan, William J ; Machielse, Bernardus N ; Kao, Mark ; Silber, Steven A ; McKew, John C ; Brewer, Carmen C ; Vite, Charles H ; Walkley, Steven U ; Austin, Christopher P ; Porter, Forbes D
    Publisher:England: Elsevier Ltd
    Journal title:The Lancet (British edition), 2017-10, Vol.390 (10104), p.1758-1768
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    Author / Creator:Hechtelt Jonker, Anneliene ; Day, Simon ; Gabaldo, Michela ; Stone, Heather ; de Kort, Martin ; O’Connor, Daniel J. ; Pasmooij, Anna Maria Gerdina
    Publisher:London: Nature Publishing Group UK
    Journal title:Nature reviews. Drug discovery, 2023-12, Vol.22 (12), p.937-938
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    Author / Creator:Low, Zheng Yao ; Farouk, Isra Ahmad ; Lal, Sunil Kumar
    Publisher:Switzerland: MDPI
    Journal title:Viruses, 2020-09, Vol.12 (9), p.1058
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    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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