Literature DB >> 18555919

Clinical trials of orphan medicines.

Brendan M Buckley1.   

Abstract

Mesh：

Year:  2008        PMID： 18555919     DOI： 10.1016/S0140-6736(08)60876-4

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


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  21 in total

1.  Development and use of new therapeutics for rare diseases: views from patients, caregivers, and advocates.

Authors:  Aaron S Kesselheim; Sarah McGraw; Lauren Thompson; Kelly O'Keefe; Joshua J Gagne
Journal:  Patient       Date:  2015-02       Impact factor: 3.883

Review 2.  Good-quality research in rare diseases: trials and tribulations.

Authors:  Davide Bolignano; Anna Pisano
Journal:  Pediatr Nephrol       Date:  2016-01-27       Impact factor: 3.714

3.  Safety-related regulatory actions for orphan drugs in the US and EU: a cohort study.

Authors:  Harald E Heemstra; Thijs J Giezen; Aukje K Mantel-Teeuwisse; Remco L A de Vrueh; Hubert G M Leufkens
Journal:  Drug Saf       Date:  2010-02-01       Impact factor: 5.606

4.  Orphan drug development is not taking off.

Authors:  Roberta Joppi; Vittorio Bertele; Silvio Garattini
Journal:  Br J Clin Pharmacol       Date:  2009-01-21       Impact factor: 4.335

Review 5.  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

6.  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

7.  Alkaptonuria: leading to the treasure in exceptions.

Authors:  Timothy M Cox
Journal:  JIMD Rep       Date:  2011-12-06

8.  Orphan therapies: making best use of postmarket data.

Authors:  Judith C Maro; Jeffrey S Brown; Gerald J Dal Pan; Lingling Li
Journal:  J Gen Intern Med       Date:  2014-08       Impact factor: 5.128

9.  When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials.

Authors:  Inmaculada de Melo-Martín; Dolan Sondhi; Ronald G Crystal
Journal:  Hum Gene Ther       Date:  2011-05-19       Impact factor: 5.695

10.  Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.

Authors:  Thomas Hundsberger; Marianne Rohrbach; Lukas Kern; Kai M Rösler
Journal:  J Neurol       Date:  2013-06-08       Impact factor: 4.849
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