AHRQ Healthcare Horizon Scanning System – Status Update

Total Page:16

File Type:pdf, Size:1020Kb

AHRQ Healthcare Horizon Scanning System – Status Update AHRQ Healthcare Horizon Scanning System – Status Update Horizon Scanning Status Update: November 2013 Prepared for: Agency for Healthcare Research and Quality U.S. Department of Health and Human Services 540 Gaither Road Rockville, MD 20850 www.ahrq.gov Contract No. HHSA290201000006C Prepared by: ECRI Institute 5200 Butler Pike Plymouth Meeting, PA 19462 November 2013 Statement of Funding and Purpose This report incorporates data collected during implementation of the Agency for Healthcare Research and Quality (AHRQ) Healthcare Horizon Scanning System by ECRI Institute under contract to AHRQ, Rockville, MD (Contract No. HHSA290201000006C). The findings and conclusions in this document are those of the authors, who are responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services. A novel intervention may not appear in this report simply because the System has not yet detected it. The list of novel interventions in the Horizon Scanning Status Update Report will change over time as new information is collected. This should not be construed as either endorsements or rejections of specific interventions. As topics are entered into the System, individual target technology reports are developed for those that appear to be closer to diffusion into practice in the United States. A representative from AHRQ served as a Contracting Officer’s Technical Representative and provided input during the implementation of the horizon scanning system. AHRQ did not directly participate in the horizon scanning, assessing the leads or topics, or provide opinions regarding potential impact of interventions. Disclaimer Regarding 508-Compliance Persons using assistive technology may not be able to fully access information in this report. For assistance contact [email protected]. Financial Disclosure Statement None of the individuals compiling this information has any affiliations or financial involvement that conflicts with the material presented in this report. Public Domain Notice This document is in the public domain and may be used and reprinted without special permission. Citation of the source is appreciated. Suggested citation: ECRI Institute. AHRQ Healthcare Horizon Scanning System Status Update. (Prepared by ECRI Institute under Contract No. HHSA290201000006C) Rockville, MD: Agency for Healthcare Research and Quality. November 2013. http://www.effectivehealthcare.ahrq.gov/reports/final.cfm. i Preface The purpose of the AHRQ Healthcare Horizon Scanning System is to conduct horizon scanning of emerging health care technologies and innovations to better inform patient-centered outcomes research investments at AHRQ through the Effective Health Care Program. The Healthcare Horizon Scanning System provides AHRQ a systematic process to identify and monitor emerging technologies and innovations in health care and to create an inventory of emerging technologies that have the highest potential for impact on clinical care, the health care system, patient outcomes, and costs. It will also be a tool for the public to identify and find information on new health care technologies and interventions. Any investigator or funder of research will be able to use the AHRQ Healthcare Horizon Scanning System to select potential topics for research. The health care technologies and innovations of interest for horizon scanning are those that have yet to diffuse into or become part of established health care practice. These health care interventions are still in the early stages of development or adoption except in the case of new applications of already-diffused technologies. Consistent with the definitions of health care interventions provided by the Institute of Medicine and the Federal Coordinating Council for Comparative Effectiveness Research, AHRQ is interested in innovations in drugs and biologics, medical devices, screening and diagnostic tests, procedures, services and programs, and care delivery. Horizon scanning involves two processes. The first is identifying and monitoring new and evolving health care interventions that are purported to or may hold potential to diagnose, treat, or otherwise manage a particular condition or to improve care delivery for a variety of conditions. The second is analyzing the relevant health care context in which these new and evolving interventions exist to understand their potential impact on clinical care, the health care system, patient outcomes, and costs. It is NOT the goal of the AHRQ Healthcare Horizon Scanning System to make predictions on the future use and costs of any health care technology. Rather, the reports will help to inform and guide the planning and prioritization of research resources. This edition of the Status Update lists interventions that have been identified and are being monitored. The next edition will be published in 2–3 months. We welcome comments on the list, which may be sent by mail to the Task Order Officer named in this report to: Agency for Healthcare Research and Quality, 540 Gaither Road, Rockville, MD 20850, or by email to: [email protected]. Richard Kronick, Ph.D. Jean Slutsky, P.A., M.S.P.H. Director Director, Center for Outcomes and Evidence Agency for Healthcare Research and Quality Agency for Healthcare Research and Quality Elise Berliner, Ph.D. Task Order Officer Center for Outcomes and Evidence Agency for Healthcare Research and Quality ii Contents Introduction ............................................................................................................................................................................. 1 Section 1. Currently Tracked Interventions: 452 Interventions .............................................................................................. 3 Table 1. AHRQ Priority Condition: 01 Arthritis and Nontraumatic Joint Disease: 14 Interventions ..................... 4 Table 2. AHRQ Priority Condition: 02 Cancer: 169 Interventions ....................................................................... 11 Table 3. AHRQ Priority Condition: 03 Cardiovascular Disease: 42 Interventions ............................................... 94 Table 4. AHRQ Priority Condition: 04 Dementia (including Alzheimer’s): 13 Interventions ............................ 116 Table 5. AHRQ Priority Condition: 05 Depression and Other Mental Health Disorders: 19 Interventions ....... 122 Table 6. AHRQ Priority Condition: 06 Developmental Delays, Attention-Deficit Hyperactivity Disorder, and Autism: 6 Interventions................................................................................................................... 132 Table 7. AHRQ Priority Condition: 07 Diabetes Mellitus: 17 Interventions ...................................................... 137 Table 8. AHRQ Priority Condition: 08 Functional Limitations and Disability: 73 Interventions ....................... 148 Table 9. AHRQ Priority Condition: 09 Infectious Disease, Including HIV-AIDS: 39 Interventions ................. 185 Table 10. AHRQ Priority Condition: 10 Obesity: 10 Interventions ...................................................................... 206 Table 11. AHRQ Priority Condition: 11 Peptic Ulcer Disease and Dyspepsia: 10 Interventions ......................... 212 Table 12. AHRQ Priority Condition: 12 Pregnancy, Including Preterm Birth: 7 Interventions ............................ 217 Table 13. AHRQ Priority Condition: 13 Pulmonary Disease, Asthma: 17 Interventions ..................................... 222 Table 14. AHRQ Priority Condition: 14 Substance Abuse: 8 Interventions ......................................................... 230 Table 15. AHRQ Priority Condition: 15 Cross-Cutting: 8 Interventions .............................................................. 234 Section 2. Interventions Added Since Last Update: 35 Interventions ................................................................................ 239 Table 16. AHRQ Priority Condition: 01 Arthritis and Nontraumatic Joint Disease: 3 Interventions ................... 240 Table 17. AHRQ Priority Condition: 02 Cancer: 14 Interventions ....................................................................... 241 Table 18. AHRQ Priority Condition: 03 Cardiovascular Disease: 0 Interventions ............................................... 248 Table 19. AHRQ Priority Condition: 04 Dementia (including Alzheimer’s): 0 Interventions .............................. 248 Table 20. AHRQ Priority Condition: 05 Depression and Other Mental Health Disorders: 1 Intervention ........... 249 Table 21. AHRQ Priority Condition: 06 Developmental Delays, Attention-Deficit Hyperactivity Disorder, and Autism: 0 Interventions................................................................................................................... 249 Table 22. AHRQ Priority Condition: 07 Diabetes Mellitus: 2 Interventions ........................................................ 250 Table 23. AHRQ Priority Condition: 08 Functional Limitations and Disability: 5 Interventions ......................... 251 Table 24. AHRQ Priority Condition: 09 Infectious Disease, Including HIV-AIDS: 8 Interventions ................... 253 Table 25. AHRQ Priority Condition: 10 Obesity: 0 Interventions ........................................................................ 256 Table 26. AHRQ Priority Condition: 11 Peptic Ulcer Disease and Dyspepsia: 0 Interventions ........................... 256 Table 27. AHRQ Priority
Recommended publications
  • Stanford Chem-H Presentation (PDF)
    KiNativ® In situ kinase profiling Stanford University ChEM-H confidential @KiNativPlatform Principle of the KiNativ platform • ATP (or ADP) acyl phosphate binds to, and covalently modifies Lysine residues in the active site • Thus, ATP acyl phosphate with a desthiobiotin tag can be used capture and quantitate kinases in a complex lysate Acyl phosphate Desthiobiotin tag ATP 2 ATP acyl phosphate probe covalently modifies kinase in the active site Lysine 2 Lysine 1 3 ATP acyl phosphate probe covalently modifies kinase in the active site Lysine 2 Lysine 1 4 Samples trypsinized, probe-labeled peptides captured with streptavidin, and analyzed by targeted LC-MS2 Identification Quantitation Explicit determination of peptide Integration of signal from MS2 sequence and probe modification site fragment ions from MS2 spectrum 5 Comprehensive Coverage of Protein and Lipid Kinases Protein kinases Atypical kinases Green: Kinases detected on KiNativ Red: Kinases not detected on KiNativ ~80% of known protein and atypical kinases identified on the platform http://www.kinativ.com/coverage/protein-lipid.html 6 Profiling compound(s) on the KiNativ platform Control sample – add probe Sample: Lysate derived from any cell line or tissue from ANY species Treated sample – add inhibitor followed by probe Inhibited kinase Green: Kinases Blue: Probe Gray: Non-kinases Red: Inhibitor 7 Profiling compound(s) on the KiNativ platform Control sample – add probe MS signalMS Sample: Lysate derived from any cell line or tissue from ANY species Treated sample – add inhibitor
    [Show full text]
  • Potential High-Impact Interventions Report Priority Area 02: Cancer
    AHRQ Healthcare Horizon Scanning System – Potential High-Impact Interventions Report Priority Area 02: Cancer Prepared for: Agency for Healthcare Research and Quality U.S. Department of Health and Human Services 540 Gaither Road Rockville, MD 20850 www.ahrq.gov Contract No. HHSA290201000006C Prepared by: ECRI Institute 5200 Butler Pike Plymouth Meeting, PA 19462 December 2012 Statement of Funding and Purpose This report incorporates data collected during implementation of the Agency for Healthcare Research and Quality (AHRQ) Healthcare Horizon Scanning System by ECRI Institute under contract to AHRQ, Rockville, MD (Contract No. HHSA290201000006C). The findings and conclusions in this document are those of the authors, who are responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services. This report’s content should not be construed as either endorsements or rejections of specific interventions. As topics are entered into the System, individual topic profiles are developed for technologies and programs that appear to be close to diffusion into practice in the United States. Those reports are sent to various experts with clinical, health systems, health administration, and/or research backgrounds for comment and opinions about potential for impact. The comments and opinions received are then considered and synthesized by ECRI Institute to identify interventions that experts deemed, through the comment process, to have potential for high impact. Please see the methods section for more details about this process. This report is produced twice annually and topics included may change depending on expert comments received on interventions issued for comment during the preceding 6 months.
    [Show full text]
  • The New Generation of Antibody Therapeutics: Current Status and Future Prospects
    Expert Intelligence for Better Decisions The New Generation of Antibody Therapeutics: Current Status and Future Prospects K. John Morrow, Jr., PhD Using This Document A navigation bar containing links to the Table of Contents, a Search feature, Insight Pharma Reports are interactive Previous/Next page arrows, and a Return button is anchored at the bottom of electronic documents which offer many each page. of the features of Web sites, including Hyperlinks throughout the document are designated by blue, underlined text. navigation, search, bookmarks, download Hyperlinks on the Index page are also rendered as blue text, and will navigate you options, and additional layered content. to a company’s Web site. Table of Contents listings are hyperlinked and will navigate you directly to that specific content, Table or Figure. Reports are also available Figures and Tables found within the document are available for download by clicking on a selected Figure or the title of a Table. The document will open in in printed format your browser. To order copies of this report, customized for l For Figures, which are in JPEG format, right-click to save to your desktop your organization, contact: l For Tables, which are in PDF format, save directly from your browser Rose LaRaia P 781-972-5444 E [email protected] References can be accessed by clicking directly on the Reference number. There is also a References page at the end of the document. Next Page Return This button will return you to a previously visited, non-sequential page Access the Table of Contents Previous Page Search the Report Insight Pharma Reports Web site Visit our site to research additional Report titles Contents Search i www.InsightPharmaReports.com Insight Pharma Reports Single User License This License permits only the Licensee with individual access to the report.
    [Show full text]
  • Refractory Early T-Cell Precursor Acute Lymphoblastic Leukemia
    Zurich Open Repository and Archive University of Zurich Main Library Strickhofstrasse 39 CH-8057 Zurich www.zora.uzh.ch Year: 2019 Venetoclax and Bortezomib in Relapsed/Refractory Early T-Cell Precursor Acute Lymphoblastic Leukemia La Starza, Roberta ; Cambò, Benedetta ; Pierini, Antonio ; Bornhauser, Beat ; Montanaro, Anna ; Bourquin, Jean-Pierre ; Mecucci, Cristina ; Roti, Giovanni DOI: https://doi.org/10.1200/PO.19.00172 Posted at the Zurich Open Repository and Archive, University of Zurich ZORA URL: https://doi.org/10.5167/uzh-198023 Journal Article Published Version The following work is licensed under a Creative Commons: Attribution 4.0 International (CC BY 4.0) License. Originally published at: La Starza, Roberta; Cambò, Benedetta; Pierini, Antonio; Bornhauser, Beat; Montanaro, Anna; Bourquin, Jean-Pierre; Mecucci, Cristina; Roti, Giovanni (2019). Venetoclax and Bortezomib in Relapsed/Refrac- tory Early T-Cell Precursor Acute Lymphoblastic Leukemia. JCO precision oncology, 3:PO.19.00172. DOI: https://doi.org/10.1200/PO.19.00172 case report Venetoclax and Bortezomib in Relapsed/ Refractory Early T-Cell Precursor Acute Lymphoblastic Leukemia Roberta La Starza, MD, PhD1; Benedetta Cambo,` MD2; Antonio Pierini, MD, PhD1; Beat Bornhauser, PhD3; Anna Montanaro2; Jean-Pierre Bourquin, MD, PhD3; Cristina Mecucci, MD, PhD1; and Giovanni Roti, MD, PhD2 INTRODUCTION CD2, CD4, CD8, and CD1a and positive for human Although in the past three decades we welcomed the leukocyte antigen (HLA-DR), CD38, CD117, CD33, advent of targeted therapies in
    [Show full text]
  • Predictive QSAR Tools to Aid in Early Process Development of Monoclonal Antibodies
    Predictive QSAR tools to aid in early process development of monoclonal antibodies John Micael Andreas Karlberg Published work submitted to Newcastle University for the degree of Doctor of Philosophy in the School of Engineering November 2019 Abstract Monoclonal antibodies (mAbs) have become one of the fastest growing markets for diagnostic and therapeutic treatments over the last 30 years with a global sales revenue around $89 billion reported in 2017. A popular framework widely used in pharmaceutical industries for designing manufacturing processes for mAbs is Quality by Design (QbD) due to providing a structured and systematic approach in investigation and screening process parameters that might influence the product quality. However, due to the large number of product quality attributes (CQAs) and process parameters that exist in an mAb process platform, extensive investigation is needed to characterise their impact on the product quality which makes the process development costly and time consuming. There is thus an urgent need for methods and tools that can be used for early risk-based selection of critical product properties and process factors to reduce the number of potential factors that have to be investigated, thereby aiding in speeding up the process development and reduce costs. In this study, a framework for predictive model development based on Quantitative Structure- Activity Relationship (QSAR) modelling was developed to link structural features and properties of mAbs to Hydrophobic Interaction Chromatography (HIC) retention times and expressed mAb yield from HEK cells. Model development was based on a structured approach for incremental model refinement and evaluation that aided in increasing model performance until becoming acceptable in accordance to the OECD guidelines for QSAR models.
    [Show full text]
  • Metabotropic Glutamate Receptors
    mGluR Metabotropic glutamate receptors mGluR (metabotropic glutamate receptor) is a type of glutamate receptor that are active through an indirect metabotropic process. They are members of thegroup C family of G-protein-coupled receptors, or GPCRs. Like all glutamate receptors, mGluRs bind with glutamate, an amino acid that functions as an excitatoryneurotransmitter. The mGluRs perform a variety of functions in the central and peripheral nervous systems: mGluRs are involved in learning, memory, anxiety, and the perception of pain. mGluRs are found in pre- and postsynaptic neurons in synapses of the hippocampus, cerebellum, and the cerebral cortex, as well as other parts of the brain and in peripheral tissues. Eight different types of mGluRs, labeled mGluR1 to mGluR8, are divided into groups I, II, and III. Receptor types are grouped based on receptor structure and physiological activity. www.MedChemExpress.com 1 mGluR Agonists, Antagonists, Inhibitors, Modulators & Activators (-)-Camphoric acid (1R,2S)-VU0155041 Cat. No.: HY-122808 Cat. No.: HY-14417A (-)-Camphoric acid is the less active enantiomer (1R,2S)-VU0155041, Cis regioisomer of VU0155041, is of Camphoric acid. Camphoric acid stimulates a partial mGluR4 agonist with an EC50 of 2.35 osteoblast differentiation and induces μM. glutamate receptor expression. Camphoric acid also significantly induced the activation of NF-κB and AP-1. Purity: ≥98.0% Purity: ≥98.0% Clinical Data: No Development Reported Clinical Data: No Development Reported Size: 10 mM × 1 mL, 100 mg Size: 10 mM × 1 mL, 5 mg, 10 mg, 25 mg (2R,4R)-APDC (R)-ADX-47273 Cat. No.: HY-102091 Cat. No.: HY-13058B (2R,4R)-APDC is a selective group II metabotropic (R)-ADX-47273 is a potent mGluR5 positive glutamate receptors (mGluRs) agonist.
    [Show full text]
  • Opportunities and Challenges for Antisense Oligonucleotide Therapies
    Received: 10 January 2020 Revised: 23 April 2020 Accepted: 8 May 2020 DOI: 10.1002/jimd.12251 REVIEW ARTICLE Opportunities and challenges for antisense oligonucleotide therapies Elsa C. Kuijper1 | Atze J. Bergsma2,3 | W.W.M. Pim Pijnappel2,3 | Annemieke Aartsma-Rus1 1Department of Human Genetics, Leiden University Medical Center, Leiden, The Abstract Netherlands Antisense oligonucleotide (AON) therapies involve short strands of modi- 2Department of Pediatrics, Center for fied nucleotides that target RNA in a sequence-specific manner, inducing Lysosomal and Metabolic Diseases, targeted protein knockdown or restoration. Currently, 10 AON therapies Erasmus Medical Center, Rotterdam, The Netherlands have been approved in the United States and Europe. Nucleotides are chem- 3Department of Clinical Genetics, Center ically modified to protect AONs from degradation, enhance bioavailability for Lysosomal and Metabolic Diseases, and increase RNA affinity. Whereas single stranded AONs can efficiently Erasmus Medical Center, Rotterdam, The Netherlands be delivered systemically, delivery of double stranded AONs requires capsulation in lipid nanoparticles or binding to a conjugate as the uptake Correspondence enhancing backbone is hidden in this conformation. With improved chem- Annemieke Aartsma-Rus, LUMC Postzone S4-P, Albinusdreef 2, 2333 ZA istry, delivery vehicles and conjugates, doses can be lowered, thereby reduc- Leiden, The Netherlands. ing the risk and occurrence of side effects. AONs can be used to knockdown Email: [email protected] or restore levels of protein. Knockdown can be achieved by single stranded Communicating Editor: Carla E. Hollak or double stranded AONs binding the RNA transcript and activating RNaseH-mediated and RISC-mediated degradation respectively. Transcript binding by AONs can also prevent translation, hence reducing protein levels.
    [Show full text]
  • Glycosylation: Rising Potential for Prostate Cancer Evaluation
    cancers Review Glycosylation: Rising Potential for Prostate Cancer Evaluation Anna Kałuza˙ * , Justyna Szczykutowicz and Mirosława Ferens-Sieczkowska Department of Chemistry and Immunochemistry, Wroclaw Medical University, Sklodowskiej-Curie 48/50, 50-369 Wroclaw, Poland; [email protected] (J.S.); [email protected] (M.F.-S.) * Correspondence: [email protected]; Tel.: +48-71-770-30-66 Simple Summary: Aberrant protein glycosylation is a well-known hallmark of cancer and is as- sociated with differential expression of enzymes such as glycosyltransferases and glycosidases. The altered expression of the enzymes triggers cancer cells to produce glycoproteins with specific cancer-related aberrations in glycan structures. Increasing number of data indicate that glycosylation patterns of PSA and other prostate-originated proteins exert a potential to distinguish between benign prostate disease and cancer as well as among different stages of prostate cancer development and aggressiveness. This review summarizes the alterations in glycan sialylation, fucosylation, truncated O-glycans, and LacdiNAc groups outlining their potential applications in non-invasive diagnostic procedures of prostate diseases. Further research is desired to develop more general algorithms exploiting glycobiology data for the improvement of prostate diseases evaluation. Abstract: Prostate cancer is the second most commonly diagnosed cancer among men. Alterations in protein glycosylation are confirmed to be a reliable hallmark of cancer. Prostate-specific antigen is the biomarker that is used most frequently for prostate cancer detection, although its lack of sensitivity and specificity results in many unnecessary biopsies. A wide range of glycosylation alterations in Citation: Kałuza,˙ A.; Szczykutowicz, prostate cancer cells, including increased sialylation and fucosylation, can modify protein function J.; Ferens-Sieczkowska, M.
    [Show full text]
  • Download Product Insert (PDF)
    PRODUCT INFORMATION Pacritinib Item No. 16709 CAS Registry No.: 937272-79-2 Formal Name: 11-[2-(1-pyrrolidinyl)ethoxy]-14,19-dioxa- 5,7,27-triazatetracyclo[19.3.1.12,6.18,12] O heptacosa-1(25),2,4,6(27),8,10,12(26), 16E,21,23-decaene Synonym: SB1518 N N H MF: C28H32N4O3 FW: 472.6 N Purity: ≥98% O Stability: ≥2 years at -20°C Supplied as: A crystalline solid N O UV/Vis.: λmax: 285 nm Laboratory Procedures For long term storage, we suggest that pacritinib​ be stored as supplied at -20°C. It should be stable for at least two years. Pacritinib is supplied as a crystalline solid. A stock solution may be made by dissolving the pacritinib in the solvent of choice. Pacritinib is soluble in the organic solvent DMSO, which should be purged with an inert gas, at a concentration of approximately 0.5 mg/ml (slightly warmed). Pacritinib is sparingly soluble in aqueous solutions. To enhance aqueous solubility, dilute the organic solvent solution into aqueous buffers or isotonic saline. If performing biological experiments, ensure the residual amount of organic solvent is insignificant, since organic solvents may have physiological effects at low concentrations. We do not recommend storing the aqueous solution for more than one day. Description FMS-like tyrosine kinase 3 (FLT3) and Janus kinase 2 (JAK2) are tyrosine kinases that mediate cytokine signaling and are frequently mutated in cancers, particularly acute myeloid leukemia.1,2 Pacritinib is an 1 inhibitor of both FLT3 and JAK2 (IC50s = 22 and 23 nM, respectively).
    [Show full text]
  • Identification of Candidate Repurposable Drugs to Combat COVID-19 Using a Signature-Based Approach
    www.nature.com/scientificreports OPEN Identifcation of candidate repurposable drugs to combat COVID‑19 using a signature‑based approach Sinead M. O’Donovan1,10, Ali Imami1,10, Hunter Eby1, Nicholas D. Henkel1, Justin Fortune Creeden1, Sophie Asah1, Xiaolu Zhang1, Xiaojun Wu1, Rawan Alnafsah1, R. Travis Taylor2, James Reigle3,4, Alexander Thorman6, Behrouz Shamsaei4, Jarek Meller4,5,6,7,8 & Robert E. McCullumsmith1,9* The COVID‑19 pandemic caused by the novel SARS‑CoV‑2 is more contagious than other coronaviruses and has higher rates of mortality than infuenza. Identifcation of efective therapeutics is a crucial tool to treat those infected with SARS‑CoV‑2 and limit the spread of this novel disease globally. We deployed a bioinformatics workfow to identify candidate drugs for the treatment of COVID‑19. Using an “omics” repository, the Library of Integrated Network‑Based Cellular Signatures (LINCS), we simultaneously probed transcriptomic signatures of putative COVID‑19 drugs and publicly available SARS‑CoV‑2 infected cell lines to identify novel therapeutics. We identifed a shortlist of 20 candidate drugs: 8 are already under trial for the treatment of COVID‑19, the remaining 12 have antiviral properties and 6 have antiviral efcacy against coronaviruses specifcally, in vitro. All candidate drugs are either FDA approved or are under investigation. Our candidate drug fndings are discordant with (i.e., reverse) SARS‑CoV‑2 transcriptome signatures generated in vitro, and a subset are also identifed in transcriptome signatures generated from COVID‑19 patient samples, like the MEK inhibitor selumetinib. Overall, our fndings provide additional support for drugs that are already being explored as therapeutic agents for the treatment of COVID‑19 and identify promising novel targets that are worthy of further investigation.
    [Show full text]
  • Genitourinary Pathology (Including Renal Tumors)
    LABORATORY INVESTIGATION THE BASIC AND TRANSLATIONAL PATHOLOGY RESEARCH JOURNAL LI VOLUME 99 | SUPPLEMENT 1 | MARCH 2019 2019 ABSTRACTS GENITOURINARY PATHOLOGY (INCLUDING RENAL TUMORS) (776-992) MARCH 16-21, 2019 PLATF OR M & 2 01 9 ABSTRACTS P OSTER PRESENTATI ONS EDUCATI ON C O M MITTEE Jason L. Hornick , C h air Ja mes R. Cook R h o n d a K. Y a nti s s, Chair, Abstract Revie w Board S ar a h M. Dr y and Assign ment Co m mittee Willi a m C. F a q ui n Laura W. La mps , Chair, C ME Subco m mittee C ar ol F. F ar v er St e v e n D. Billi n g s , Interactive Microscopy Subco m mittee Y uri F e d ori w Shree G. Shar ma , Infor matics Subco m mittee Meera R. Ha meed R aj a R. S e et h al a , Short Course Coordinator Mi c h ell e S. Hir s c h Il a n W ei nr e b , Subco m mittee for Unique Live Course Offerings Laksh mi Priya Kunju D a vi d B. K a mi n s k y ( Ex- Of ici o) A n n a M ari e M ulli g a n Aleodor ( Doru) Andea Ri s h P ai Zubair Baloch Vi nita Parkas h Olca Bast urk A nil P ar w a ni Gregory R. Bean , Pat h ol o gist-i n- Trai ni n g D e e p a P atil D a ni el J.
    [Show full text]
  • The Use of Ataluren in the Effective Management of Duchenne Muscular Dystrophy
    Review Neuromuscular Diseases Early Diagnosis and Treatment – The Use of Ataluren in the Effective Management of Duchenne Muscular Dystrophy Eugenio Mercuri,1 Ros Quinlivan2 and Sylvie Tuffery-Giraud3 1. Catholic University, Rome, Italy; 2. Great Ormond Street Hospital and National Hospital for Neurology and Neurosurgery, London, UK; 3. Laboratory of Genetics of Rare Diseases (LGMR), University of Montpellier, Montpellier, France DOI: https://doi.org/10.17925/ENR.2018.13.1.31 he understanding of the natural history of Duchenne muscular dystrophy (DMD) is increasing rapidly and new treatments are emerging that have the potential to substantially improve the prognosis for patients with this disabling and life-shortening disease. For many, Thowever, there is a long delay between the appearance of symptoms and DMD diagnosis, which reduces the possibility of successful treatment. DMD results from mutations in the large dystrophin gene of which one-third are de novo mutations and two-thirds are inherited from a female carrier. Roughly 75% of mutations are large rearrangements and 25% are point mutations. Certain deletions and nonsense mutations can be treated whereas many other mutations cannot currently be treated. This emphasises the need for early genetic testing to identify the mutation, guide treatment and inform genetic counselling. Treatments for DMD include corticosteroids and more recently, ataluren has been approved in Europe, the first disease-modifying therapy for treating DMD caused by nonsense mutations. The use of ataluren in DMD is supported by positive results from phase IIb and phase III studies in which the treatment produced marked improvements in the 6-minute walk test, timed function tests such as the 10 m walk/run test and the 4-stair ascent/descent test compared with placebo.
    [Show full text]