Literature DB >> 27245396

Worldwide collaboration for orphan drug designation.

Segundo Mariz1, James H Reese2, Kerstin Westermark3, Lesley Greene4, Takahiro Goto5, Tatsuro Hoshino5, Jordi Llinares-Garcia2, Bruno Sepodes6.   

Abstract

Mesh：

Year:  2016        PMID： 27245396     DOI： 10.1038/nrd.2016.80

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


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  5 in total

Review 1.  Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.

Authors:  M Miles Braun; Sheiren Farag-El-Massah; Kui Xu; Timothy R Coté
Journal:  Nat Rev Drug Discov       Date:  2010-06-07       Impact factor: 84.694

2.  Kiran Mazumdar-Shaw.

Authors:  Kiran Mazumdar-Shaw
Journal:  Nat Rev Drug Discov       Date:  2010-07       Impact factor: 84.694

Review 3.  European regulation on orphan medicinal products: 10 years of experience and future perspectives.

Authors:  Kerstin Westermark; Birthe Byskov Holm; Mirjam Söderholm; Jordi Llinares-Garcia; Frida Rivière; Stiina Aarum; Florence Butlen-Ducuing; Stelios Tsigkos; Agnieszka Wilk-Kachlicka; Cinzia N'Diamoi; János Borvendég; David Lyons; Bruno Sepodes; Brigitte Bloechl-Daum; André Lhoir; Mariana Todorova; Ioannis Kkolos; Kateřina Kubáčková; Heidrun Bosch-Traberg; Vallo Tillmann; Veijo Saano; Emmanuel Héron; Rembert Elbers; Miranda Siouti; Judit Eggenhofer; Patrick Salmon; Maurizio Clementi; Dainis Krieviņš; Aušra Matulevičiene; Henri Metz; Albert Cilia Vincenti; Albertha Voordouw; Bożenna Dembowska-Bagińska; Ana Corrêa Nunes; Flavia Mirela Saleh; Tatiana Foltánová; Martin Možina; Josep Torrent i Farnell; Björn Beerman; Segundo Mariz; Marie Pauline Evers; Lesley Greene; Sigurdur Thorsteinsson; Lars Gramstad; Maria Mavris; Fabrizia Bignami; Annie Lorence; Chantal Belorgey
Journal:  Nat Rev Drug Discov       Date:  2011-05       Impact factor: 84.694

Review 4.  Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives.

Authors:  Peipei Song; Jianjun Gao; Yoshinori Inagaki; Norihiro Kokudo; Wei Tang
Journal:  Intractable Rare Dis Res       Date:  2012-02

5.  Establishing medical plausibility in the context of orphan medicines designation in the European Union.

Authors:  Stelios Tsigkos; Segundo Mariz; Jordi Llinares; Laura Fregonese; Stiina Aarum; Frauke Naumann-Winter; Naumann-Winter Frauke; Kerstin Westermark; Bruno Sepodes
Journal:  Orphanet J Rare Dis       Date:  2014-12-05       Impact factor: 4.123
  5 in total
  14 in total

Review 1.  From promising molecules to orphan drugs: Early clinical drug development.

Authors:  Marc Dooms
Journal:  Intractable Rare Dis Res       Date:  2017-02

Review 2.  Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.

Authors:  Erik Tambuyzer; Benjamin Vandendriessche; Christopher P Austin; Philip J Brooks; Kristina Larsson; Katherine I Miller Needleman; James Valentine; Kay Davies; Stephen C Groft; Robert Preti; Tudor I Oprea; Marco Prunotto
Journal:  Nat Rev Drug Discov       Date:  2019-12-13       Impact factor: 84.694

Review 3.  Opportunities and Challenges in Drug Development for Pediatric Cancers.

Authors:  Theodore W Laetsch; Steven G DuBois; Julia Glade Bender; Margaret E Macy; Lucas Moreno
Journal:  Cancer Discov       Date:  2020-12-04       Impact factor: 38.272

Review 4.  Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective.

Authors:  Christopher P Austin; Christine M Cutillo; Lilian P L Lau; Anneliene H Jonker; Ana Rath; Daria Julkowska; David Thomson; Sharon F Terry; Béatrice de Montleau; Diego Ardigò; Virginie Hivert; Kym M Boycott; Gareth Baynam; Petra Kaufmann; Domenica Taruscio; Hanns Lochmüller; Makoto Suematsu; Carlo Incerti; Ruxandra Draghia-Akli; Irene Norstedt; Lu Wang; Hugh J S Dawkins
Journal:  Clin Transl Sci       Date:  2017-10-23       Impact factor: 4.689

Review 5.  Progress in Rare Diseases Research 2010-2016: An IRDiRC Perspective.

Authors:  Hugh J S Dawkins; Ruxandra Draghia-Akli; Paul Lasko; Lilian P L Lau; Anneliene H Jonker; Christine M Cutillo; Ana Rath; Kym M Boycott; Gareth Baynam; Hanns Lochmüller; Petra Kaufmann; Yann Le Cam; Virginie Hivert; Christopher P Austin
Journal:  Clin Transl Sci       Date:  2017-10-23       Impact factor: 4.689

6.  Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.

Authors:  Kim Pauwels; Isabelle Huys; Minne Casteels; Kristina Larsson; Caroline Voltz; Karri Penttila; Thomas Morel; Steven Simoens
Journal:  Orphanet J Rare Dis       Date:  2017-02-16       Impact factor: 4.123

Review 7.  Lithium's antiviral effects: a potential drug for CoViD-19 disease?

Authors:  Andrea Murru; Mirko Manchia; Tomas Hajek; René E Nielsen; Janusz K Rybakowski; Gabriele Sani; Thomas G Schulze; Leonardo Tondo; Michael Bauer
Journal:  Int J Bipolar Disord       Date:  2020-05-20

Review 8.  Regulatory Standards in Orphan Medicinal Product Designation in the EU.

Authors:  Stelios Tsigkos; Segundo Mariz; Maria Elzbieta Sheean; Kristina Larsson; Armando Magrelli; Violeta Stoyanova-Beninska
Journal:  Front Med (Lausanne)       Date:  2021-06-25

9.  The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact.

Authors:  Hanns Lochmüller; Josep Torrent I Farnell; Yann Le Cam; Anneliene H Jonker; Lilian Pl Lau; Gareth Baynam; Petra Kaufmann; Hugh Js Dawkins; Paul Lasko; Christopher P Austin; Kym M Boycott
Journal:  Eur J Hum Genet       Date:  2017-11-20       Impact factor: 4.246

Review 10.  Glioblastoma Treatments: An Account of Recent Industrial Developments.

Authors:  Edouard Alphandéry
Journal:  Front Pharmacol       Date:  2018-09-13       Impact factor: 5.810
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