Orphan drugs : future viability of current forecasting models, in light of impending changes to influential market factors [PDF]
, 2011 Thesis (S.M.)--Harvard-MIT Division of Health Sciences and Technology, 2011.Cataloged from PDF version of thesis.Includes bibliographical references (p. 58-61).Interviews were conducted to establish a baseline for how orphan drug forecasting is currently
Gottlieb, Joshua
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Feasibility of following up gamma-hydroxybutyric acid concentrations in sodium oxybate (Xyrem®)-treated narcoleptic patients using dried blood spot sampling at home : an exploratory study [PDF]
, 2013 Background: Gamma-hydroxybutyric acid (GHB), well known as a party drug, especially in Europe, is also legally used (sodium oxybate, Xyrem (R)) to treat a rare sleep disorder, narcolepsy with cataplexy.
Hertegonne, Katrien +3 more
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Cannabinoids in the treatment of epilepsy: current status and future prospects [PDF]
, 2020 Cannabidiol (CBD) is one of the prominent phytocannabinoids found in Cannabis sativa, differentiating from Δ9-tetrahydrocannabinol (THC) for its non-intoxicating profile and its antianxiety/antipsychotic effects.
Albini M. +6 more
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Deletion of Gpr27 in vivo reduces insulin mRNA but does not result in diabetes. [PDF]
, 2020 Gpr27 is a highly conserved, orphan G protein coupled receptor (GPCR) previously implicated in pancreatic beta cell insulin transcription and glucose-stimulated insulin secretion in vitro. Here, we characterize a whole-body mouse knockout of Gpr27. Gpr27
Chopra, Deeksha G +4 more
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Orphan drug: Development trends and strategies
Journal of Pharmacy and Bioallied Sciences, 2010 The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will loose their exclusivity in
Aarti Sharma +3 more
doaj +1 more source
Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act [PDF]
With substantial fixed costs of drug development, more common conditions can support more products. If additional pharmaceutical products are beneficial, they will attract greater consumption and promote better health, e.g. greater longevity.
Frank R. Lichtenberg, Joel Waldfogel
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Functional classification of G-Protein coupled receptors, based on their specific ligand coupling patterns [PDF]
, 2006 Functional identification of G-Protein Coupled Receptors (GPCRs) is one of the current focus areas of pharmaceutical research. Although thousands of GPCR sequences are known, many of them re- main as orphan sequences (the activating ligand is unknown ...
Bakır, Burcu, Sezerman, Uğur
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Prediction and classification for GPCR sequences based on ligand specific features [PDF]
, 2006 Functional identification of G-Protein Coupled Receptors (GPCRs) is one of the current focus areas of pharmaceutical research. Although thousands of GPCR sequences are known, many of them are orphan sequences (the activating ligand is unknown). Therefore,
F. Horn +8 more
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Do investors value the FDA orphan drug designation?
Orphanet Journal of Rare Diseases, 2017 Background The Orphan Drug Act is an important piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases.
Kathleen L. Miller
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Three classes of ligands each bind to distinct sites on the orphan G protein-coupled receptor GPR84 [PDF]
, 2017 Medium chain fatty acids can activate the pro-inflammatory receptor GPR84 but so also can molecules related to 3,3′-diindolylmethane. 3,3′-Diindolylmethane and decanoic acid acted as strong positive allosteric modulators of the function of each other and
De Vos, Steve +8 more
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