Orphan drugs and the NHS: Should we value rarity [PDF]
, 2005 Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients.
Claxton, K., McCabe, C., Tsuchiya, A.
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Pharmaceutical compounding of orphan active ingredients in Belgium : how community and hospital pharmacists can address the needs of patients with rare diseases [PDF]
, 2019 Background: Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages.
Boussery, Koen +7 more
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Physiopathological Implications of 7TM Receptors [PDF]
, 2010 Seven-transmembrane (7TM) receptors are one of the most important proteins involved in perception of extracellular stimuli and regulation of variety of intracellular signaling pathways.
Cygankiewicz, Adam
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Functional profiles of orphan membrane transporters in the life cycle of the malaria parasite [PDF]
, 2016 Assigning function to orphan membrane transport proteins and prioritizing candidates for detailed biochemical characterization remain fundamental challenges and are particularly important for medically relevant pathogens, such as malaria parasites.
Kenthirapalan, Sanketha +3 more
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Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC
Hepatology, EarlyView., 2022 Abstract Background and Aims Type 3 innate lymphoid cells (ILC3s) are essential for host defense against infection and tissue homeostasis. However, their role in the development of HCC has not been adequately confirmed. In this study, we investigated the immunomodulatory role of short‐chain fatty acids (SCFAs) derived from intestinal microbiota in ILC3
Chupeng Hu +11 more
wiley +1 more source
Nuclear receptors in vascular biology [PDF]
, 2015 Nuclear receptors sense a wide range of steroids and hormones (estrogens, progesterone, androgens, glucocorticoid, and mineralocorticoid), vitamins (A and D), lipid metabolites, carbohydrates, and xenobiotics.
A Chawla +56 more
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The Promising Viral Threat to Bacterial Resistance: The Uncertain Patentability of Phage Therapeutics and the Necessity of Alternative Incentives [PDF]
, 2014 Bacteriophages, or “phages,” are a category of highly adept and adaptable viruses that can infect and kill bacteria. With concerns over the burgeoning antibiotic-resistance crisis looming in recent years, scientists and policymakers have expressed a ...
Todd, Kelly
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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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G protein-coupled receptor 35: an emerging target in inflammatory and cardiovascular disease [PDF]
, 2015 G protein-coupled receptor 35 (GPR35) is an orphan receptor, discovered in 1998, that has garnered interest as a potential therapeutic target through its association with a range of diseases.
Amanda E Mackenzie +4 more
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Angewandte Chemie, EarlyView.Human synenkephalin [1–53] (hSYN), an analogue peptide of shrew saliva neurotoxins, was synthesized and its structural characteristics studied. Synthetic hSYN potently activated the T‐type voltage‐gated Ca channel hCav3.2 but did not paralyze mealworms. These findings offer new insight into neurological disorder treatment and evolutionary mechanisms of
Ryo Fukuoka +5 more
wiley +2 more sources