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https://www.readbyqxmd.com/read/28218743/vitamin-d-signaling-and-melanoma-role-of-vitamin-d-and-its-receptors-in-melanoma-progression-and-management
#1
REVIEW
Andrzej T Slominski, Anna A Brożyna, Michal A Zmijewski, Wojciech Jóźwicki, Anton M Jetten, Rebecca S Mason, Robert C Tuckey, Craig A Elmets
Ultraviolet B (UVB), in addition to having carcinogenic activity, is required for the production of vitamin D3 (D3) in the skin which supplies >90% of the body's requirement. Vitamin D is activated through hydroxylation by 25-hydroxylases (CYP2R1 or CYP27A1) and 1α-hydroxylase (CYP27B1) to produce 1,25(OH)2D3, or through the action of CYP11A1 to produce mono-di- and trihydroxy-D3 products that can be further modified by CYP27B1, CYP27A1, and CYP24A1. The active forms of D3, in addition to regulating calcium metabolism, exert pleiotropic activities, which include anticarcinogenic and anti-melanoma effects in experimental models, with photoprotection against UVB-induced damage...
February 20, 2017: Laboratory Investigation; a Journal of Technical Methods and Pathology
https://www.readbyqxmd.com/read/28213288/inhibition-of-estrogen-related-receptor-%C3%AE-attenuates-vascular-smooth-muscle-cell-proliferation-and-migration-by-regulating-rhoa-p27-kip1-and-%C3%AE-catenin-wnt4-signaling-pathway
#2
Qunyi Li, Lei Zhu, Liudi Zhang, Haifei Chen, Yingfeng Zhu, Yongli Du, Wanxian Zhong, Mingkang Zhong, Xiaojin Shi
RhoA/p27(Kip1) and β-Catenin/Wnt4 signaling processes play central roles in proliferation and migration in vascular smooth muscle cells (VSMCs). ERRα, a member of orphan nuclear receptors, is a potent prognostic factor in breast, ovarian, colon and other types of tumors. However, biological significance of ERRα in VSMCs as well as the molecular mechanisms remains largely unknown. Therefore, the present study was designed to investigate whether ERRα is involved in the proliferation and migration of VSMCs in vitro and neointimal formation in vivo...
February 14, 2017: European Journal of Pharmacology
https://www.readbyqxmd.com/read/28209382/err%C3%AE-a-junior-orphan-with-a-senior-role-in-metabolism
#3
REVIEW
Jagannath Misra, Don-Kyu Kim, Hueng-Sik Choi
Estrogen-related receptor (ERR)γ is an orphan nuclear hormone receptor that belongs to the ERR subfamily of transcription factors. No endogenous ligand has been identified to date. ERRγ possesses ligand-independent transcriptional activity that is regulated by co-regulator interactions, and post-translational modifications (PTMs). Recent data from animal models have established ERRγ as a crucial mediator of multiple endocrine and metabolic signals. ERRγ plays important roles in pathological conditions such as insulin resistance, alcoholic liver injury, and cardiac hypertrophy, and controls energy metabolism in the heart, skeletal muscle, and pancreatic β cells...
February 13, 2017: Trends in Endocrinology and Metabolism: TEM
https://www.readbyqxmd.com/read/28203051/a-review-and-update-on-orphan-drugs-for-the-treatment-of-noninfectious-uveitis
#4
REVIEW
Caiyun You, Haitham F Sahawneh, Lina Ma, Buraa Kubaisi, Alexander Schmidt, C Stephen Foster
INTRODUCTION: Uveitis, a leading cause of preventable blindness around the world, is a critically underserved disease in regard to the medications approved for use. Multiple immunomodulatory therapy (IMT) drugs are appropriate for uveitis therapy but are still off-label. These IMT agents, including antimetabolites, calcineurin inhibitors, alkylating agents, and biologic agents, have been designated as "orphan drugs" and are widely used for systemic autoimmune diseases or organ transplantation...
2017: Clinical Ophthalmology
https://www.readbyqxmd.com/read/28197652/-intravascular-papillary-endothelial-hyperplasia-ipeh-masson-s-pseudoangiosarcoma-on-the-forefoot
#5
P Rottler, A Wilke, T M Frangen, C Kuhnen, B Schley
Intravascular papillary endothelial hyperplasia (IPEH)/Masson's pseudoangiosarcoma is a rare (orphan) disease of the forefoot that has not yet been described in Europe. The pathoanatomic examination of a young patient with a vague interdigital space-consuming lesion revealed a intravascular papillary endothelial hyperplasia/Masson's pseudoangiosarcoma. The intravascular papillary endothelial hyperplasia can be mainly detected in the upper limbs as well as cervically. Heretofore, the intravascular papillary endothelial hyperplasia has only been described in Japan and the USA...
February 14, 2017: Der Orthopäde
https://www.readbyqxmd.com/read/28195755/prioritizing-rare-diseases
#6
Johanna Wiss, Lars-Ake Levin, David Andersson, Gustav Tinghög
BACKGROUND: Measuring societal preferences for rarity has been proposed to determine whether paying premium prices for orphan drugs is acceptable. OBJECTIVE: To investigate societal preferences for rarity and how psychological factors affect such preferences. METHOD: A postal survey containing resource allocation dilemmas involving patients with a rare disease and patients with a common disease, equal in severity, was sent out to a randomly selected sample of the population in Sweden (return rate 42...
February 1, 2017: Medical Decision Making: An International Journal of the Society for Medical Decision Making
https://www.readbyqxmd.com/read/28190666/design-of-a-framework-for-the-deployment-of-collaborative-independent-rare-disease-centric-registries-gaucher-disease-registry-model
#7
Matthew I Bellgard, Kathryn R Napier, Alan H Bittles, Jeffrey Szer, Sue Fletcher, Nikolajs Zeps, Adam A Hunter, Jack Goldblatt
Orphan drug clinical trials often are adversely affected by a lack of high quality treatment efficacy data that can be reliably compared across large patient cohorts derived from multiple governmental and country jurisdictions. It is critical that these patient data be captured with limited corporate involvement. For some time, there have been calls to develop collaborative, non-proprietary, patient-centric registries for post-market surveillance of aspects related to orphan drug efficacy. There is an urgent need for the development and sustainable deployment of these 'independent' registries that can capture comprehensive clinical, genetic and therapeutic information on patients with rare diseases...
January 27, 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28183254/metastatic-colorectal-cancer-role-of-target-therapies-and-future-perspectives
#8
Anna Nappi, Massimiliano Berretta, Carmela Romano, Salvatore Tafuto, Antonino Cassata, Rossana Casaretti, Lucrezia Silvestro, Chiara De Divitiis, Lara Alessandrini, Francesco Fiorica, Alessandro Ottaiano, Guglielmo Nasti
Today, we are experiencing a real cultural revolution in the therapeutic approach to cancer of the colon - rectum, that, by orphan disease, it is now becoming an important paradigm of scientific innovations and concepts. Survival of patients with metastatic colorectal cancer (m-CRC) has been significantly improved with the introduction of the monoclonal antibodies that have as target the vascular endothelial growth factor (VEGF) and the epidermal growth factor receptor (EGFR). Novel agents such as aflibercept and regorafenib have recently been approved...
February 8, 2017: Current Cancer Drug Targets
https://www.readbyqxmd.com/read/28161253/carbonic-anhydrases-from-trypanosoma-and-leishmania-as-anti-protozoan-drug-targets
#9
REVIEW
Alane B Vermelho, Giseli R Capaci, Igor A Rodrigues, Verônica S Cardoso, Ana Maria Mazotto, Claudiu T Supuran
Trypanosoma cruzi and Leishmania spp. are protozoa of the Trypanosomatidae family, being the etiological agents of two widespread parasitic diseases, Chagas disease and leishmaniasis, respectively. Both parasites are the focus of worldwide research with the aim to find effective and less toxic drugs than the few ones available so far, and for controlling the spread of the diseases. Carbonic anhydrases (CAs, EC 4.2.1.1) belonging to the α- and β-class were recently identified in these protozoans and several studies suggested that they could be new targets for drug development...
January 24, 2017: Bioorganic & Medicinal Chemistry
https://www.readbyqxmd.com/read/28161117/new-drugs-for-rare-diseases-in-children
#10
Klaus Rose
PURPOSE: United States (US) Pediatric Legislation (PL) was introduced in 1997 to improve children's health. The European Union PL (EUPL) has been in force since 2007. Both PLs facilitate additional pediatric research on primarily adult drugs. The EUPL declares that the forces of the market are not sufficient for children. Without a pediatric investigation plan, new drugs can no longer be registered with the European Union. New ways on how to facilitate drug development for rare pediatric diseases are being proposed...
February 1, 2017: Clinical Therapeutics
https://www.readbyqxmd.com/read/28160756/receptor-tyrosine-kinase-like-orphan-receptor-1-ror-1-an-emerging-target-for-diagnosis-and-therapy-of-chronic-lymphocytic-leukemia
#11
REVIEW
Leili Aghebati-Maleki, Mahdi Shabani, Behzad Baradaran, Morteza Motallebnezhad, Jafar Majidi, Mehdi Yousefi
Chronic lymphocytic leukemia (CLL) is characterized by reposition of malignant B cells in the blood, bone marrow, spleen and lymph nodes. It remains the most common leukemia in the Western world. Within the recent years, major breakthroughs have been made to prolong the survival and improve the health of patients. Despite these advances, CLL is still recognized as a disease without definitive cure. New treatment approaches, based on unique targets and novel drugs, are highly desired for CLL therapy. The Identification and subsequent targeting of molecules that are overexpressed uniquely in malignant cells not normal ones play critical roles in the success of anticancer therapeutic strategies...
February 1, 2017: Biomedicine & Pharmacotherapy, Biomédecine & Pharmacothérapie
https://www.readbyqxmd.com/read/28160230/risk-evaluation-and-mitigation-strategies-remss-are-they-improving-drug-safety-a-critical-review-of-remss-requiring-elements-to-assure-safe-use-etasu
#12
REVIEW
Pol F Boudes
Risk Evaluation and Mitigation Strategies (REMSs) with Elements to Assure Safe Use (ETASU) are requested for drugs with significant safety risks. We reviewed REMS programs issued since 2011 to evaluate their rationales, characteristics, and consistencies, and evaluated their impact on improving drug safety. We conducted a literature search and a survey of relevant websites (FDA, manufacturers, and REMSs). ETASU characteristics were summarized. REMS risks were compared with labeled risks, including black box warnings...
February 3, 2017: Drugs in R&D
https://www.readbyqxmd.com/read/28152950/brentuximab-vedotin-in-relapsed-refractory-hodgkin-lymphoma-following-autologous-stem-cell-transplant-a-cost-effectiveness-analysis-updated-with-5-year-follow-up-data-from-the-pivotal-trial
#13
Gemma Kay, Esprit Ma, Beth Woods, James Eaton, Peter Sajosi, Andreas Engstrom, Ross Selby, Eugene Benson, Jeremy Teasell, Akshara Richhariya, Andrew Briggs, Vijayveer Bonthapally
: 19 Background: In 2015, the Scottish Medicines Consortium (SMC) made a positive recommandation for brentuximab vedotin (BV) in patients with relapsed or refractory (R/R) Hodgkin lymphoma (HL) who have received autologous stem cell transplantation (ASCT) based on 3-year follow-up data from the pivotal phase 2 single-arm trial (SG035-0003; NCT00848926). At 3-years, the incremental cost-effectiveness ratio (ICER) for brentuximab vedotin compared with chemotherapy +/- radiotherapy (C/R) was £43,731 per quality-adjusted life year (QALY)...
March 2016: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28141737/anaesthesia-and-orphan-disease-difficult-ventilation-following-intubation-in-goldenhar-syndrome
#14
Faiz Hasham, Noud van Helmond, Richard Sidlow
No abstract text is available yet for this article.
March 2017: European Journal of Anaesthesiology
https://www.readbyqxmd.com/read/28141736/anaesthesia-and-orphan-diseases-airway-management-in-proteus-disease
#15
Anastasia Alevizou, Panagiota Florou, Chrysanti Batistaki, Georgia Kostopanagiotou
No abstract text is available yet for this article.
March 2017: European Journal of Anaesthesiology
https://www.readbyqxmd.com/read/28141735/anaesthesia-and-orphan-disease-a-child-with-congenital-cataract-facial-dysmorphism-neuropathy-ccfdn-syndrome-a-case-report
#16
Oliver W Masters, Elonka Bergmans, Karl-Christian Thies
No abstract text is available yet for this article.
March 2017: European Journal of Anaesthesiology
https://www.readbyqxmd.com/read/28141734/anaesthesia-and-orphan-disease-rapid-sequence-induction-in-systemic-mastocytosis
#17
Christoph Unterbuchner, Marina Hierl, Timo Seyfried, Thomas Metterlein
No abstract text is available yet for this article.
March 2017: European Journal of Anaesthesiology
https://www.readbyqxmd.com/read/28140549/treatment-options-for-pyoderma-gangrenosum
#18
REVIEW
Sven R Quist, Luise Kraas
Pyoderma gangrenosum (PG) is an orphan disease. While research on such disorders is based on only few randomized multicenter as well as retrospective studies, most of the data comes from case series of small patient groups. Apart from topical and intralesional therapeutic options for early stages and mild disease courses, treatment predominantly involves systemic therapeutic agents. Besides systemic corticosteroids and cyclosporine A (CsA), options also include intravenous immunoglobulins (IVIG) and biologics such as the TNFα inhibitors infliximab, adalimumab, and etanercept; the interleukin (IL) 12/23 antibody ustekinumab; the IL-1 receptor antagonist anakinra; and the IL-1β antibody canakinumab...
January 2017: Journal der Deutschen Dermatologischen Gesellschaft, Journal of the German Society of Dermatology: JDDG
https://www.readbyqxmd.com/read/28140542/therapieoptionen-beim-pyoderma-gangraenosum
#19
REVIEW
Sven R Quist, Luise Kraas
Das Pyoderma gangraenosum (PG) gehört zu den orphan diseases, deren Erforschung sich lediglich auf einzelne, randomisierte, multizentrische sowie retrospektive Studien stützen kann und überwiegend auf Fallserien an kleinen Patientenkollektiven beruht. Die Therapie basiert neben topischen und lokal intraläsionalen Therapieoptionen, bei initialem und leichtem Krankheitsverlauf, insbesondere auf der Gabe von Systemtherapeutika. Diese beinhaltet neben den systemischen Glukokortikosteroiden und Ciclosporin A (CsA) auch Biologika wie intravenöses Immunglobulin G (IVIG), die TNFα-Inhibitoren Infliximab, Adalimumab und Etanercept, den IL-12/23-Antikörper Ustekinumab, den Interleukin-1-Rezeptorantagonist Anakinra und den Interleukin-1β-Antikörper Canakinumab...
January 2017: Journal der Deutschen Dermatologischen Gesellschaft, Journal of the German Society of Dermatology: JDDG
https://www.readbyqxmd.com/read/28137218/metabolic-and-cardiovascular-actions-of-nesfatin-1-implications-in-health-and-disease
#20
Naresh Ramesh, Kavishankar Gawli, Venkatakiran Pasupulleti, Suraj Unniappan
BACKGROUND: Metabolic homeostasis is achieved by proper functioning of a complex endocrine milieu, comprising of signals arising from the brain and peripheral tissues. Our knowledge of the factors regulating such balance is rapidly evolving, as new signaling molecules are characterized. Of central interest is nesfatin-1, owing to its multifunctional tissue specific actions regulating food intake, body weight, blood glucose and cardiac functions. METHOD: This review discusses the tissue/system wide distribution and actions of nesfatin-1 in regulating metabolic and cardiovascular functions in healthy conditions and diseases...
January 30, 2017: Current Pharmaceutical Design
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