Literature DB >> 18555916

Does orphan drug legislation really answer the needs of patients?

Marlene E Haffner1, Josep Torrent-Farnell, Paul D Maher.   

Abstract

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Year:  2008        PMID： 18555916     DOI： 10.1016/S0140-6736(08)60873-9

Source DB:  PubMed          Journal:  Lancet        ISSN： 0140-6736            Impact factor:   79.321


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  17 in total

1.  Orphan diseases: state of the drug discovery art.

Authors:  Claude-Henry Volmar; Claes Wahlestedt; Shaun P Brothers
Journal:  Wien Med Wochenschr       Date:  2016-01-27

Review 2.  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

3.  Access to orphan drugs despite poor quality of clinical evidence.

Authors:  Alain G Dupont; Philippe B Van Wilder
Journal:  Br J Clin Pharmacol       Date:  2011-04       Impact factor: 4.335

Review 4.  [Orphan drugs from the perspective of the Drug Commission of the German Medical Association : Opportunities and challenges].

Authors:  Wolf-Dieter Ludwig
Journal:  Internist (Berl)       Date:  2019-04       Impact factor: 0.743

Review 5.  Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).

Authors:  Markus Ries
Journal:  J Inherit Metab Dis       Date:  2017-03-17       Impact factor: 4.982

6.  Orphan drug policies and use in pediatric nephrology.

Authors:  Diana Karpman; Peter Höglund
Journal:  Pediatr Nephrol       Date:  2016-10-13       Impact factor: 3.714

Review 7.  Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease.

Authors:  Elizabeth A Ottinger; Mark L Kao; Nuria Carrillo-Carrasco; Nicole Yanjanin; Roopa Kanakatti Shankar; Marjo Janssen; Marcus Brewster; Ilona Scott; Xin Xu; Jim Cradock; Pramod Terse; Seameen J Dehdashti; Juan Marugan; Wei Zheng; Lili Portilla; Alan Hubbs; William J Pavan; John Heiss; Charles H Vite; Steven U Walkley; Daniel S Ory; Steven A Silber; Forbes D Porter; Christopher P Austin; John C McKew
Journal:  Curr Top Med Chem       Date:  2014       Impact factor: 3.295

8.  Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering-A Cross-Sectional Analysis.

Authors:  Clemens Stockklausner; Anette Lampert; Georg F Hoffmann; Markus Ries
Journal:  Oncologist       Date:  2016-03-28

9.  Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

Authors:  Anne E M Brabers; Ellen H M Moors; Sonja van Weely; Remco L A de Vrueh
Journal:  Orphanet J Rare Dis       Date:  2011-09-05       Impact factor: 4.123

10.  The prevalence and cost of unapproved uses of top-selling orphan drugs.

Authors:  Aaron S Kesselheim; Jessica A Myers; Daniel H Solomon; Wolfgang C Winkelmayer; Raisa Levin; Jerry Avorn
Journal:  PLoS One       Date:  2012-02-21       Impact factor: 3.240
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