Results 11 to 20 of about 22,199 (155)
Frontiers in Immunology, 2021 Small heterodimer partner interacting leucine zipper protein (SMILE) is an orphan nuclear receptor and a member of the bZIP family of proteins. We investigated the mechanism by which SMILE suppressed the development of inflammatory bowel disease (IBD ...
SeungCheon Yang +16 more
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Gene and Cell Therapies Overview Under the Light of Health Economics [PDF]
Health Economics and Management Review, 2022 With the increase in drug development studies for rare diseases, gene therapies have recently come to the fore more frequently. In addition to orphan drugs used in the treatment of rare diseases, advanced therapy medicinal products have been developed ...
Ekin Begum Karahan, Guvenc Kockaya
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Small heterodimer partner-targeting therapy inhibits systemic inflammatory responses through mitochondrial uncoupling protein 2. [PDF]
PLoS ONE, 2013 The orphan nuclear receptor, small heterodimer partner (SHP), appears to play a negative regulatory role in innate immune signaling. Emerging evidence warrants further study on the therapeutic targeting of SHP to suppress excessive and deleterious ...
Chul-Su Yang +8 more
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Is the Orphan Drug Industry the Proper Scapegoat for Unethical Research Funding?
Voices in Bioethics, 2019 The mainstream media and general public have expressed frustration in recent months with the confluence of capitalism and the pharmaceutical research and development enterprise.
Michael Menconi
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Pharmaceuticals, 2023 The iron chelating orphan drug deferiprone (L1), discovered over 40 years ago, has been used daily by patients across the world at high doses (75–100 mg/kg) for more than 30 years with no serious toxicity.
George J. Kontoghiorghes
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Frontiers in Pharmacology, 2023 Introduction: In 2017 the drug chenodeoxycholic acid (CDCA) became unavailable to Dutch patients with the rare inborn error of metabolism cerebrotendinous xanthomatosis (CTX).
Natalja Bouwhuis +4 more
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Frontiers in Microbiology, 2022 Natural products from microorganisms are important sources for drug discovery. With the development of high-throughput sequencing technology and bioinformatics, a large amount of uncharacterized biosynthetic gene clusters (BGCs) in microorganisms have ...
Xingxing Li +10 more
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Cryo‐EM structure of orphan G protein‐coupled receptor GPR21
MedComm, 2023 GPR21 belongs to class A orphan G protein‐coupled receptor (GPCR). The endogenous ligands for human GPR21 remain unidentified. GPR21 expression is associated with developing type 2 diabetes (T2DM), a multifactorial metabolic disease caused by pancreatic ...
Thian‐Sze Wong +8 more
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Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group) [PDF]
Frontiers in Pharmacology, 2021 Today policy makers face the challenge to devise a policy framework that improves orphan medicinal product (OMP) development by creating incentives to deliver treatments where there are none and to authorize innovative and transformative treatments where treatments already exist. The European Expert Group on Orphan Drug Incentives (hereafter, OD Expert
Aartsma-Rus, Annemieke +2 more
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Oftalʹmologiâ, 2022 Neurotrophic keratopathy (NTK) is an orphan, polyetiological disease that has recently been identified more frequently. Drug-resistant stages of NTC often require urgent surgical treatment.
M. Ezugbaya +7 more
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