Book of Abstracts, GTH, Bremen 2020;
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Supplementary Materials
Supplementary materials Supplementary Table S1: MGNC compound library Ingredien Molecule Caco- Mol ID MW AlogP OB (%) BBB DL FASA- HL t Name Name 2 shengdi MOL012254 campesterol 400.8 7.63 37.58 1.34 0.98 0.7 0.21 20.2 shengdi MOL000519 coniferin 314.4 3.16 31.11 0.42 -0.2 0.3 0.27 74.6 beta- shengdi MOL000359 414.8 8.08 36.91 1.32 0.99 0.8 0.23 20.2 sitosterol pachymic shengdi MOL000289 528.9 6.54 33.63 0.1 -0.6 0.8 0 9.27 acid Poricoic acid shengdi MOL000291 484.7 5.64 30.52 -0.08 -0.9 0.8 0 8.67 B Chrysanthem shengdi MOL004492 585 8.24 38.72 0.51 -1 0.6 0.3 17.5 axanthin 20- shengdi MOL011455 Hexadecano 418.6 1.91 32.7 -0.24 -0.4 0.7 0.29 104 ylingenol huanglian MOL001454 berberine 336.4 3.45 36.86 1.24 0.57 0.8 0.19 6.57 huanglian MOL013352 Obacunone 454.6 2.68 43.29 0.01 -0.4 0.8 0.31 -13 huanglian MOL002894 berberrubine 322.4 3.2 35.74 1.07 0.17 0.7 0.24 6.46 huanglian MOL002897 epiberberine 336.4 3.45 43.09 1.17 0.4 0.8 0.19 6.1 huanglian MOL002903 (R)-Canadine 339.4 3.4 55.37 1.04 0.57 0.8 0.2 6.41 huanglian MOL002904 Berlambine 351.4 2.49 36.68 0.97 0.17 0.8 0.28 7.33 Corchorosid huanglian MOL002907 404.6 1.34 105 -0.91 -1.3 0.8 0.29 6.68 e A_qt Magnogrand huanglian MOL000622 266.4 1.18 63.71 0.02 -0.2 0.2 0.3 3.17 iolide huanglian MOL000762 Palmidin A 510.5 4.52 35.36 -0.38 -1.5 0.7 0.39 33.2 huanglian MOL000785 palmatine 352.4 3.65 64.6 1.33 0.37 0.7 0.13 2.25 huanglian MOL000098 quercetin 302.3 1.5 46.43 0.05 -0.8 0.3 0.38 14.4 huanglian MOL001458 coptisine 320.3 3.25 30.67 1.21 0.32 0.9 0.26 9.33 huanglian MOL002668 Worenine -
Justification
Justification to the Resolution of the Federal Joint Committee (G-BA) on an Amendment of the Pharmaceuticals Directive (AM ‑ RL): Annex XII – Resolutions on the Benefit Assessment of Medicinal Products with New Active Ingredients in Accordance with Section 35a SGB V Damoctocog alfa pegol From 20 June 2019 Contents 1. Legal basis ................................................................................................................ 2 2. Key points of the resolution ..................................................................................... 2 2.1 Additional benefit of the medicinal product in relation to the appropriate comparator therapy ..................................................................................................... 3 2.1.1 Approved therapeutic indication of damoctocog alfa pegol (Jivi®) in accordance with the summary of product characteristics ............................................ 3 2.1.2 Appropriate comparator therapy ................................................................... 3 2.1.3 Extent and probability of the additional benefit .............................................. 5 2.1.4 Summary of the assessment ........................................................................ 6 2.2 Number of patients or demarcation of patient groups eligible for treatment ...... 6 2.3 Requirements for a quality-assured application ................................................ 7 2.4 Treatment costs .............................................................................................. -
Pan-Proteomic Approaches to Diseases
ANNUAL MEETING ABSTRACTS 433A were Grade 2. The CD45 labeling indices for Grade 1 and 2 tumors were 7.4 ± 19.7% of cyst fluid tumor marker is variable. Recently, molecular tests of cyst fluid have been and 4.7 ± 11.1% (p=0.47). The Ki-67+/CD45+ labeling indices for Grade 1 and 2 tumors applied and show high specificity in the differentiation of the pancreatic cystic lesions. were 0.0029 ± 0.10% and 0.60 ± 3.6% (p=0.45). In this study, we investigated the usefulness of molecular analysis of pancreatic cyst Conclusions: These results suggest that tumor-infiltrating lymphocytes did not affect fluid in the setting of inconclusive cytology and non-contributory tumor markers test. the Ki-67 labeling index in this series of PanNETs, and will require validation with a Design: We retrospectively reviewed pancreatic cystic lesions with EUS-FNA cytology, larger patient population. cyst fluid tumor markers (CEA and amylase) and molecular tests (K-ras mutation and loss of heterozygocity) from 2009-2011. The inconclusive cytology diagnosis was 1802 MicroRNAs as Diagnostic Markers for Pancreatic Ductal defined as “non-diagnostic” or “atypical cytology”. Non-contributory cyst fluid chemical Adenocarcinoma and Pancreatic Intraepithelial Neoplasm analysis includes 2 situations: 1) amylase<250U/L and CEA <192 ng/ml or 2) amylase >250 U/L and CEA >192ng/ml. The molecular tests and the histopathologic results of Y Xue, AN Abou Tayoun, KM Abo, JM Pipas, SR Gordon, TB Gardner, RJ Barth, AA the resection specimens were analyzed. Suriawinata, GJ Tsongalis. Dartmouth-Hitchcock Medical Center, Lebanon, NH. -
Download Special Issue
BioMed Research International Integrated Analysis of Multiscale Large-Scale Biological Data for Investigating Human Disease 2016 Guest Editors: Tao Huang, Lei Chen, Jiangning Song, Mingyue Zheng, Jialiang Yang, and Zhenguo Zhang Integrated Analysis of Multiscale Large-Scale Biological Data for Investigating Human Disease 2016 BioMed Research International Integrated Analysis of Multiscale Large-Scale Biological Data for Investigating Human Disease 2016 GuestEditors:TaoHuang,LeiChen,JiangningSong, Mingyue Zheng, Jialiang Yang, and Zhenguo Zhang Copyright © 2016 Hindawi Publishing Corporation. All rights reserved. This is a special issue published in “BioMed Research International.” All articles are open access articles distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Contents Integrated Analysis of Multiscale Large-Scale Biological Data for Investigating Human Disease 2016 Tao Huang, Lei Chen, Jiangning Song, Mingyue Zheng, Jialiang Yang, and Zhenguo Zhang Volume 2016, Article ID 6585069, 2 pages New Trends of Digital Data Storage in DNA Pavani Yashodha De Silva and Gamage Upeksha Ganegoda Volume 2016, Article ID 8072463, 14 pages Analyzing the miRNA-Gene Networks to Mine the Important miRNAs under Skin of Human and Mouse Jianghong Wu, Husile Gong, Yongsheng Bai, and Wenguang Zhang Volume 2016, Article ID 5469371, 9 pages Differential Regulatory Analysis Based on Coexpression Network in Cancer Research Junyi -
Active Data Protection Period Register of Innovative Drugs
Register Of Innovative Drugs Products for Human Use - Active Data Protection Period Notice of Drug(s) Containing Pediatric Submission Compliance 6 Year "No Data Protection Medicinal Ingredient(s)* Innovative Drug Manufacturer the Medicinal Extension Number Date File" Date Ends Ingredient / Variations Yes / No yyyy-mm-dd abemaciclib 215268 Verzenio Eli Lilly Canada Inc. N/A 2019-04-08 2025-04-08 N/A 2027-04-08 acalabrutinib 214504 Calquence AstraZeneca Canada Inc. N/A 2019-08-23 2025-08-23 N/A 2027-08-23 aclidinium bromide 157598 Tudorza Genuair AstraZeneca Canada Inc. Duaklir Genuair 2013-07-29 2019-07-29 N/A 2021-07-29 afatinib dimaleate 158730 Giotrif Boehringer Ingelheim (Canada) Ltd. N/A 2013-11-01 2019-11-01 N/A 2021-11-01 aflibercept 149321 Eylea Bayer Inc. N/A 2013-11-08 2019-11-08 N/A 2021-11-08 albiglutide 165145 Eperzan GlaxoSmithKline Inc. N/A 2015-07-15 2021-07-15 N/A 2023-07-15 alectinib hydrochloride 189442 Alecensaro Hoffmann-La Roche Limited N/A 2016-09-29 2022-09-29 N/A 2024-09-29 alirocumab 183116 Praluent Sanofi-aventis Canada Inc. N/A 2016-04-11 2022-04-11 N/A 2024-04-11 alogliptin benzoate 158335 Nesina Takeda Canada Inc. Kazano 2013-11-27 2019-11-27 N/A 2021-11-27 Oseni anthrax antigen filtrate 212387 Biothrax Emergent Biodefense Operations N/A 2018-12-13 2024-12-13 N/A 2026-12-13 Lansing LLC anthrax immune globulin (human) 200446 Anthrasil Emergent BioSolutions Canada Inc. N/A 2017-11-06 2023-11-06 Yes 2026-05-06 antihemophilic factor (recombinant 163447 Eloctate Bioverativ Canada Inc. -
Regulation of the Cortical Actin Cytoskeleton in Budding Yeast by Twinfilin, a Ubiquitous Actin Monomer-Sequestering Protein Bruce L
Regulation of the Cortical Actin Cytoskeleton in Budding Yeast by Twinfilin, a Ubiquitous Actin Monomer-sequestering Protein Bruce L. Goode, David G. Drubin, and Pekka Lappalainen Department of Molecular and Cell Biology, University of California, Berkeley, California 94720-3202 Abstract. Here we describe the identification of a cells, a green fluorescent protein (GFP)–twinfilin fu- novel 37-kD actin monomer binding protein in budding sion protein localizes primarily to cytoplasm, but also to yeast. This protein, which we named twinfilin, is com- cortical actin patches. Overexpression of the twinfilin posed of two cofilin-like regions. In our sequence data- gene (TWF1) results in depolarization of the cortical base searches we also identified human, mouse, and actin patches. A twf1 null mutation appears to result in Caenorhabditis elegans homologues of yeast twinfilin, increased assembly of cortical actin structures and is suggesting that twinfilins form an evolutionarily con- synthetically lethal with the yeast cofilin mutant cof1- served family of actin-binding proteins. Purified recom- 22, shown previously to cause pronounced reduction in binant twinfilin prevents actin filament assembly by turnover of cortical actin filaments. Taken together, forming a 1:1 complex with actin monomers, and inhib- these results demonstrate that twinfilin is a novel, its the nucleotide exchange reaction of actin monomers. highly conserved actin monomer-sequestering protein Despite the sequence homology with the actin filament involved in regulation of the cortical actin cytoskeleton. depolymerizing cofilin/actin-depolymerizing factor (ADF) proteins, our data suggests that twinfilin does Key words: actin • cytoskeleton • budding yeast • not induce actin filament depolymerization. In yeast twinfilin • cofilin HE actin cytoskeleton plays an essential role in mul- aments. -
Stembook 2018.Pdf
The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances FORMER DOCUMENT NUMBER: WHO/PHARM S/NOM 15 WHO/EMP/RHT/TSN/2018.1 © World Health Organization 2018 Some rights reserved. This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0 IGO licence (CC BY-NC-SA 3.0 IGO; https://creativecommons.org/licenses/by-nc-sa/3.0/igo). Under the terms of this licence, you may copy, redistribute and adapt the work for non-commercial purposes, provided the work is appropriately cited, as indicated below. In any use of this work, there should be no suggestion that WHO endorses any specific organization, products or services. The use of the WHO logo is not permitted. If you adapt the work, then you must license your work under the same or equivalent Creative Commons licence. If you create a translation of this work, you should add the following disclaimer along with the suggested citation: “This translation was not created by the World Health Organization (WHO). WHO is not responsible for the content or accuracy of this translation. The original English edition shall be the binding and authentic edition”. Any mediation relating to disputes arising under the licence shall be conducted in accordance with the mediation rules of the World Intellectual Property Organization. Suggested citation. The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances. Geneva: World Health Organization; 2018 (WHO/EMP/RHT/TSN/2018.1). Licence: CC BY-NC-SA 3.0 IGO. Cataloguing-in-Publication (CIP) data. -
Hemophilia Treatment: Factors to Consider
11/2/2020 Disclosures Hemophilia Treatment: ▫ There are no relevant financial interests to disclose for Factors to Consider myself or my spouse/partner from within the last 12 months. PJ Barker, PharmD, BCPPS Clinical Coordinator St. Jude Children’s Research Hospital 1 2 Objectives Hemophilia Hemophilia A Hemophilia B • Discuss standard half life and extended half life • Factor VIII (FVIII) Deficiency • Factor IX (FIX) Deficiency factor treatment options for patients with • 80% of cases • 20% of cases hemophilia • Describe emicizumab-kxwh role in therapy for Hemophilia A MedlinePlus, National Library of Medicine. 3 4 Complications and Classification Factor fVIII fIX Concentrates Severity Mild Moderate Severe Bypassing Agents rfVIIa aPCC Activated prothrombin Baseline Factor (BPA) Factor 7 activated complex concentrate Level (% of 6-30% 1-5% <1 % normal) Tranexamic Aminocaproic Antifibrinolytics acid acid Major Minor No Bleeding Trauma Trauma Trauma Emicizumab- • Life threatening bleeds • Inhibitors (factor antibodies) Desmopressin kxwh • Joint damage and • Viral Transmission Options Treatment Immobility Srivastava A. Hemophilia. 2020. 5 6 1 11/2/2020 Treatment Approaches Prophylactic Therapy • Primary Prophylaxis Mild ▫ Initiation of preventative therapy before onset of joint Episodic Treatment disease, second clinically relevant bleed, and 3 years of Moderate age • Secondary Prophylaxis ▫ Initiation of preventative therapy after two or more joint bleeds but before disease and typically > 3 years of age Prophylaxis Episodic • Tertiary -
Molecular Characterization of Mutant Mouse Strains Generated from the EUCOMM/KOMP-CSD ES Cell Resource
Mamm Genome DOI 10.1007/s00335-013-9467-x Molecular Characterization of Mutant Mouse Strains Generated from the EUCOMM/KOMP-CSD ES Cell Resource Edward Ryder • Diane Gleeson • Debarati Sethi • Sapna Vyas • Evelina Miklejewska • Priya Dalvi • Bishoy Habib • Ross Cook • Matthew Hardy • Kalpesh Jhaveri • Joanna Bottomley • Hannah Wardle-Jones • James N. Bussell • Richard Houghton • Jennifer Salisbury • William C. Skarnes • Sanger Mouse Genetics Project • Ramiro Ramirez-Solis Received: 3 April 2013 / Accepted: 27 June 2013 Ó The Author(s) 2013. This article is published with open access at Springerlink.com Abstract The Sanger Mouse Genetics Project generates Introduction knockout mice strains using the EUCOMM/KOMP-CSD embryonic stem (ES) cell collection and characterizes the The extensive genetic resources available for the mouse, consequences of the mutations using a high-throughput including the sequencing and annotation of the genomes of primary phenotyping screen. Upon achieving germline multiple inbred laboratory strains (Church et al. 2009; transmission, new strains are subject to a panel of quality Keane et al. 2011; Flicek et al. 2012; Wong et al. 2012), control (QC) PCR- and qPCR-based assays to confirm the have facilitated comprehensive comparisons with the correct targeting, cassette structure, and the presence of the human genome (Guigo et al. 2003; Zheng-Bradley et al. 30 LoxP site (required for the potential conditionality of the 2010; Mouse ENCODE Consortium et al. 2012). This allele). We report that over 86 % of the 731 strains studied makes the mouse a powerful tool for both investigating showed the correct targeting and cassette structure, of gene function and modelling disease progression in mam- which 97 % retained the 30 LoxP site. -
UNIVERSITY of CALIFORNIA, SAN DIEGO Measuring
UNIVERSITY OF CALIFORNIA, SAN DIEGO Measuring and Correlating Blood and Brain Gene Expression Levels: Assays, Inbred Mouse Strain Comparisons, and Applications to Human Disease Assessment A dissertation submitted in partial satisfaction of the requirements for the degree of Doctor of Philosophy in Biomedical Sciences by Mary Elizabeth Winn Committee in charge: Professor Nicholas J Schork, Chair Professor Gene Yeo, Co-Chair Professor Eric Courchesne Professor Ron Kuczenski Professor Sanford Shattil 2011 Copyright Mary Elizabeth Winn, 2011 All rights reserved. 2 The dissertation of Mary Elizabeth Winn is approved, and it is acceptable in quality and form for publication on microfilm and electronically: Co-Chair Chair University of California, San Diego 2011 iii DEDICATION To my parents, Dennis E. Winn II and Ann M. Winn, to my siblings, Jessica A. Winn and Stephen J. Winn, and to all who have supported me throughout this journey. iv TABLE OF CONTENTS Signature Page iii Dedication iv Table of Contents v List of Figures viii List of Tables x Acknowledgements xiii Vita xvi Abstract of Dissertation xix Chapter 1 Introduction and Background 1 INTRODUCTION 2 Translational Genomics, Genome-wide Expression Analysis, and Biomarker Discovery 2 Neuropsychiatric Diseases, Tissue Accessibility and Blood-based Gene Expression 4 Mouse Models of Human Disease 5 Microarray Gene Expression Profiling and Globin Reduction 7 Finding and Accessible Surrogate Tissue for Neural Tissue 9 Genetic Background Effect Analysis 11 SPECIFIC AIMS 12 ENUMERATION OF CHAPTERS -
(INN) for Biological and Biotechnological Substances
WHO/EMP/RHT/TSN/2019.1 International Nonproprietary Names (INN) for biological and biotechnological substances (a review) 2019 WHO/EMP/RHT/TSN/2019.1 International Nonproprietary Names (INN) for biological and biotechnological substances (a review) 2019 International Nonproprietary Names (INN) Programme Technologies Standards and Norms (TSN) Regulation of Medicines and other Health Technologies (RHT) Essential Medicines and Health Products (EMP) International Nonproprietary Names (INN) for biological and biotechnological substances (a review) FORMER DOCUMENT NUMBER: INN Working Document 05.179 © World Health Organization 2019 All rights reserved. Publications of the World Health Organization are available on the WHO website (www.who.int) or can be purchased from WHO Press, World Health Organization, 20 Avenue Appia, 1211 Geneva 27, Switzerland (tel.: +41 22 791 3264; fax: +41 22 791 4857; e-mail: [email protected]). Requests for permission to reproduce or translate WHO publications –whether for sale or for non-commercial distribution– should be addressed to WHO Press through the WHO website (www.who.int/about/licensing/copyright_form/en/index.html). The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of the World Health Organization concerning the legal status of any country, territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries. Dotted and dashed lines on maps represent approximate border lines for which there may not yet be full agreement. The mention of specific companies or of certain manufacturers’ products does not imply that they are endorsed or recommended by the World Health Organization in preference to others of a similar nature that are not mentioned. -
EHL FVIII Regimens for the Management of Hemophilia A
PRACTICE AID EHL FVIII Regimens for the Management of Hemophilia A Current and Emerging Extended Half-Life Factor VIII Therapies Approved for Hemophilia A Investigational Damoctocog alfa pegol Rurioctocog alfa pegol BIVV001 Efmoroctocog alfa Turoctocog alfa pegol FDA-Approved Extended Half-Life Products for Hemophilia A1-5 Damoctocog Efmoroctocog Rurioctocog Turoctocog Alfa Pegol Alfa Alfa Pegol Alfa Pegol 2018 Adults and 2014 2015 2019 FDA Approval adolescents, Adults and children Adults and children Adults and children previously treated rFVIII Design B-domain deleted B-domain deleted Full length B-domain truncated Modification to PEG PEG PEG Fc fusion EHL (60 kDa) (20 kDa) (40 kDa) Half-Life, h (adult) 19 19 14.3 - 16 19 30-40 units/kg 50 units/kg 2x/week every 4 days 50 units/kg every 4 days Adjust: 45-60 units/kg 40-50 units/kg Adjust: less or more Dosing (adult) every 5 days; 2x/week frequent dosing Adjust: 25-65 units/kg may be further based on bleeding every 3-5 days adjusted to less or episodes more frequent dosing • All are highly effective when used as prophylaxis with ABRs significantly Efficacy improved compared to no prophylaxis • Also effective for breakthrough bleeds and perioperative management Safety • Generally well tolerated with no unexpected safety issues Access the activity, “Implementing Care With Extended Half-Life Factor VIII Therapy in Hemophilia A: An Interdisciplinary Conversation on Personalized Patient Management,” at PeerView.com/CEV40 PRACTICE AID EHL FVIII Regimens for the Management of Hemophilia