Literature DB >> 21081597

The problem of orphan drugs.

Robin E Ferner, Dyfrig A Hughes.   

Abstract

Mesh：

Year:  2010        PMID： 21081597     DOI： 10.1136/bmj.c6456

Source DB:  PubMed          Journal:  BMJ        ISSN： 0959-8138


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  7 in total

1.  Orphan drugs for rare diseases: is it time to revisit their special market access status?

Authors:  Steven Simoens; David Cassiman; Marc Dooms; Eline Picavet
Journal:  Drugs       Date:  2012-07-30       Impact factor: 9.546

2.  Orphan drug policies and use in pediatric nephrology.

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

3.  Drugs for rare disorders.

Authors:  Serge Cremers; Jeffrey K Aronson
Journal:  Br J Clin Pharmacol       Date:  2017-06-27       Impact factor: 4.335

4.  Pricing and reimbursement of orphan drugs: the need for more transparency.

Authors:  Steven Simoens
Journal:  Orphanet J Rare Dis       Date:  2011-06-17       Impact factor: 4.123

5.  Aggregated N-of-1 trials for unlicensed medicines for small populations: an assessment of a trial with ephedrine for myasthenia gravis.

Authors:  Stephanie S Weinreich; Charlotte Vrinten; Marja R Kuijpers; Alexander F Lipka; Kirsten J M Schimmel; Erik W van Zwet; Christine Gispen-de Wied; Yechiel A Hekster; Jan J G M Verschuuren; Martina C Cornel
Journal:  Orphanet J Rare Dis       Date:  2017-05-12       Impact factor: 4.123

6.  Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU.

Authors:  Kerstin Noëlle Vokinger; Aaron S Kesselheim
Journal:  BMJ Open       Date:  2019-10-10       Impact factor: 2.692

Review 7.  The Role of Zinc in the Treatment of Wilson's Disease.

Authors:  Abolfazl Avan; Anna Członkowska; Susan Gaskin; Alberto Granzotto; Stefano L Sensi; Tjaard U Hoogenraad
Journal:  Int J Mol Sci       Date:  2022-08-18       Impact factor: 6.208
  7 in total

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