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Literature DB >> 22460117

Rare diseases and orphan drugs.

Irena Melnikova.

Abstract

Mesh：

Year: 2012 PMID： 22460117 DOI： 10.1038/nrd3654

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

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2 in total

Review 1. Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Authors: Erik Tambuyzer
Journal: Nat Rev Drug Discov Date: 2010-11-09 Impact factor: 84.694

2. Orphan drug pricing and payer management in the United States: are we approaching the tipping point?

Authors: Rebecca Hyde; Diana Dobrovolny
Journal: Am Health Drug Benefits Date: 2010-01

2 in total

52 in total

1. Responsibility and intellectual property in synthetic biology: A proposal for using Responsible Research and Innovation as a basic framework for intellectual property decisions in synthetic biology.

Authors: Harald König; Pedro Dorado-Morales; Manuel Porcar
Journal: EMBO Rep Date: 2015-08-12 Impact factor: 8.807

2. Clinical trials in rare disease: challenges and opportunities.

Authors: Erika F Augustine; Heather R Adams; Jonathan W Mink
Journal: J Child Neurol Date: 2013-09 Impact factor: 1.987

Review 3. [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 4. Drug discovery and development for rare genetic disorders.

Authors: Wei Sun; Wei Zheng; Anton Simeonov
Journal: Am J Med Genet A Date: 2017-07-21 Impact factor: 2.802

5. The discovery of medicines for rare diseases.

Authors: David C Swinney; Shuangluo Xia
Journal: Future Med Chem Date: 2014-06 Impact factor: 3.808

Review 6. Drug discovery effectiveness from the standpoint of therapeutic mechanisms and indications.

Authors: Hsin-Pei Shih; Xiaodan Zhang; Alex M Aronov
Journal: Nat Rev Drug Discov Date: 2017-10-27 Impact factor: 84.694

Review 7. Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges.

Authors: Antonello Di Paolo; Elena Arrigoni
Journal: Drugs Date: 2018-03 Impact factor: 9.546

Review 8. Modeling rare diseases with induced pluripotent stem cell technology.

Authors: Ruthellen H Anderson; Kevin R Francis
Journal: Mol Cell Probes Date: 2018-01-05 Impact factor: 2.365

Review 9. iPSC modeling of rare pediatric disorders.

Authors: Bethany A Freel; Jordan N Sheets; Kevin R Francis
Journal: J Neurosci Methods Date: 2019-12-04 Impact factor: 2.390

10. Strategy for identifying repurposed drugs for the treatment of cerebral cavernous malformation.

Authors: Christopher C Gibson; Weiquan Zhu; Chadwick T Davis; Jay A Bowman-Kirigin; Aubrey C Chan; Jing Ling; Ashley E Walker; Luca Goitre; Simona Delle Monache; Saverio Francesco Retta; Yan-Ting E Shiu; Allie H Grossmann; Kirk R Thomas; Anthony J Donato; Lisa A Lesniewski; Kevin J Whitehead; Dean Y Li
Journal: Circulation Date: 2014-12-08 Impact factor: 29.690