Results 21 to 30 of about 68,610 (290)

Cryo‐EM structure of orphan G protein‐coupled receptor GPR21

open access: yesMedComm, 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

G protein-coupled receptors not currently in the spotlight: free fatty acid receptor 2 and GPR35 [PDF]

open access: yes, 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
core   +1 more source

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]

open access: yesFrontiers 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

Lamellar Keratoplasty as a Method of Treatment Patients with Neurotrophic Keratopathy II and III Stages Outcome

open access: yesOftalʹmologiâ, 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

Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

open access: yesOrphanet 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]

open access: yesPLoS 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]

open access: yesCell 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

The Promising Viral Threat to Bacterial Resistance: The Uncertain Patentability of Phage Therapeutics and the Necessity of Alternative Incentives [PDF]

open access: yes, 2019
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
core   +2 more sources

Estrogen-related receptor α (ERRα) : a novel target in type 2 diabetes [PDF]

open access: yes, 2005
Recent studies have shown that reduced mitochondrial content and function in skeletal muscle are common features of type 2 diabetes. Here, we review the molecular mechanisms involved in the regulation of mitochondrial genes in skeletal muscle, focusing ...
Handschin, C., Mootha, V. K.
core   +1 more source

Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC

open access: yesHepatology, 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

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