Results 21 to 30 of about 68,732 (285)
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
doaj +1 more source
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
doaj +1 more source
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
doaj +1 more source
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
core +3 more sources
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
openaire +4 more sources
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
doaj +1 more source
Orphanet Journal of Rare Diseases, 2018 Background To assess uncertainty in regulatory decision-making for orphan medicinal products (OMP), a summary of the current basis for approval is required; a systematic grouping of medical conditions may be useful in summarizing information and issuing ...
Caridad Pontes +13 more
doaj +1 more source
Identification of Tetraazacyclic Compounds as Novel Potent Inhibitors Antagonizing RORγt Activity and Suppressing Th17 Cell Differentiation. [PDF]
PLoS ONE, 2015 CD4+ T-helper cells that produce interleukin-17 (Th17 cells) are characterized as pathological T-helper cells in autoimmune diseases. Differentiation of human and mouse Th17 cells requires a key transcription regulator, retinoic acid receptor-related ...
Qingfeng Ding +4 more
doaj +1 more source
The Orphan Drug Act and the Development of Stem Cell-Based Products for Rare Diseases [PDF]
Cell Stem Cell, 2010 The Orphan Drug Act encourages the development of products for rare diseases and conditions. Many conditions that stand to benefit from stem cell-based products are rare diseases. We address the Orphan Drug Act in relation to the development of stem cell-based products.
Freeman, Scott N. +3 more
openaire +2 more sources
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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