Access to Life-Saving Medicines and Intellectual Property Rights: An Ethical Assessment [PDF]
, 2011 © 2011 Cambridge University Press. Online edition of the journal is available at http://journals.cambridge.org/action/displayJournal?jid=CQHDying before one’s time has been a prominent theme in classic literature and poetry.
Aquinas +20 more
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Citation: 'orphan drug' in the IUPAC Compendium of Chemical Terminology, 5th ed.; International Union of Pure and Applied Chemistry; 2025. Online version 5.0.0, 2025. 10.1351/goldbook.12699 • License: The IUPAC Gold Book is licensed under Creative Commons Attribution-ShareAlike CC BY-SA 4.0 International for individual terms.
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Race, Money and Medicines [PDF]
, 2006 Taking notice of race is both risky and inevitable, in medicine no less than in other endeavors. The literature on race as a classifying tool in clinical research poses this core dilemma: On the one hand, race can be a useful stand-in for unstudied ...
Bloche, Maxwell Gregg
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Understanding Disease Heterogeneity and Patient Characteristics in Patients with Amyotrophic Lateral Sclerosis (ALS) [PDF]
, 2019 Background: Amytrophic lateral sclerosis (ALS) is a fatal neurologic disease that is projected to double in worldwide incidence in the next 20 years.
Mathew, Veena
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Unraveling the functional role of the orphan solute carrier, SLC22A24 in the transport of steroid conjugates through metabolomic and genome-wide association studies. [PDF]
, 2019 Variation in steroid hormone levels has wide implications for health and disease. The genes encoding the proteins involved in steroid disposition represent key determinants of interindividual variation in steroid levels and ultimately, their effects ...
Boerwinkle, Eric +13 more
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BMJ Open, 2019 Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).Design and setting Identification of all cancer drugs (initial or ...
Aaron S Kesselheim +1 more
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G protein-coupled receptors not currently in the spotlight: free fatty acid receptor 2 and GPR35 [PDF]
, 2017 It is widely appreciated that G protein-coupled receptors have been the most successfully exploited class of targets for the development of small molecule medicines. Despite this, to date, less than 15% of the non-olfactory G protein-coupled receptors in
Milligan, Graeme
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Promoting Academic Entrepreneurship in Europe and the United States: Creating an Intellectual Property Regime to Facilitate the Efficient Transfer of Knowledge from the Lab to the Patient [PDF]
, 2015 In 2014, the European Commission announced the launch of a study of knowledge transfer by public research organizations and other institutes of higher learning “to determine which additional measures might be needed to ensure an optimal flow of knowledge
Bagley, Constance E. +1 more
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Are payers treating orphan drugs differently?
Journal of Market Access & Health Policy, 2014 Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years.
Joshua P. Cohen, Abigail Felix
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Drugs for exceptionally rare diseases: a commentary on Hughes et al [PDF]
, 2006 Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and ...
Claxton, K. +3 more
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