Literature DB >> 21119719

Accelerating orphan drug development.

Timothy R Coté1, Kui Xu, Anne R Pariser.   

Abstract

Interest in developing drugs for rare diseases has increased substantially in recent years. This article from the US Food and Drug Administration highlights the role of regulators in catalysing further progress in this field.

Mesh:

Year:  2010        PMID: 21119719     DOI: 10.1038/nrd3340

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


  1 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

  1 in total
  8 in total

Review 1.  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

Review 2.  Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants.

Authors:  Jane P F Bai; Jeffrey S Barrett; Gibert J Burckart; Bernd Meibohm; Hari Cheryl Sachs; Lynne Yao
Journal:  AAPS J       Date:  2013-01-19       Impact factor: 4.009

Review 3.  Drug repositioning for personalized medicine.

Authors:  Yvonne Y Li; Steven Jm Jones
Journal:  Genome Med       Date:  2012-03-30       Impact factor: 11.117

4.  Some Numbers behind Canada's Decision to Adopt an Orphan Drug Policy: US Orphan Drug Approvals in Canada, 1997-2012.

Authors:  Matthew Herder; Timothy Mark Krahn
Journal:  Healthc Policy       Date:  2016-05

5.  The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases - recommendations of the IRDiRC Data Mining and Repurposing Task Force.

Authors:  Noel T Southall; Madhusudan Natarajan; Lilian Pek Lian Lau; Anneliene Hechtelt Jonker; Benoît Deprez; Tim Guilliams; Lawrence Hunter; Carin Ma Rademaker; Virginie Hivert; Diego Ardigò
Journal:  Orphanet J Rare Dis       Date:  2019-10-15       Impact factor: 4.123

Review 6.  Innovative Study Designs Optimizing Clinical Pharmacology Research in Infants and Children.

Authors:  Stephen J Balevic; Michael Cohen-Wolkowiez
Journal:  J Clin Pharmacol       Date:  2018-10       Impact factor: 2.860

7.  Why the Shift? Taking a Closer Look at the Growing Interest in Niche Markets and Personalized Medicine.

Authors:  Shannon Gibson; Hamid R Raziee; Trudo Lemmens
Journal:  World Med Health Policy       Date:  2015-03

Review 8.  Clinical studies in lysosomal storage diseases: Past, present, and future.

Authors:  Pol F Boudes
Journal:  Rare Dis       Date:  2013-10-07
  8 in total

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