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INSMED INC FORM 10-K (Annual Report) Filed 03/16/10 for the Period Ending 12/31/09 Address 8720 STONY POINT PARKWAY SUITE 200 RICHMOND, VA 23235 Telephone 804-565-3000 CIK 0001104506 Symbol INSM SIC Code 2834 - Pharmaceutical Preparations Industry Biotechnology & Drugs Sector Healthcare Fiscal Year 12/31 http://www.edgar-online.com © Copyright 2010, EDGAR Online, Inc. All Rights Reserved. Distribution and use of this document restricted under EDGAR Online, Inc. Terms of Use. UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended: December 31, 2009 OR TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________ to __________ Commission File Number 0-30739 INSMED INCORPORATED (Exact name of registrant as specified in its charter) Virginia 54 -1972729 (State or other jurisdiction of incorporation or organization) (I.R.S. employer identification no.) 8720 Stony Point Parkway Richmond, Virginia 23235 (804) 565 -3000 (Address of principal executive offices) (Registrant ’s telephone number including area code) Securities registered pursuant to Section 12(b) of the Act: Title of each class Name of each exchange on which registered Common Stock, par value $0.01/share Nasdaq Capital Market Securities registered pursuant to Section 12(g) of the Act: None Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. -
Table of Contents
Therapeutic systems for Insulin-like growth factor-I Dissertation zur Erlangung des naturwissenschaftlichen Doktorgrades der Julius-Maximilians-Universität Würzburg vorgelegt von Isabel Schultz aus Dahn Würzburg 2015 Eingereicht bei der Fakultät für Chemie und Pharmazie am Gutachter der schriftlichen Arbeit 1. Gutachter: 2. Gutachter: Prüfer des öffentlichen Promotionskolloquiums 1. Prüfer: 2. Prüfer: 3. Prüfer: Datum des öffentlichen Promotionskolloquiums Doktorurkunde ausgehändigt am TABLE OF CONTENTS TABLE OF CONTENTS SUMMARY ............................................................................... 1 ZUSAMMEMFASSUNG .......................................................... 5 CHAPTER I ............................................................................... 9 DRUG DELIVERY OF INSULIN-LIKE GROWTH FACTOR I CHAPTER II ............................................................................ 45 INSULIN-LIKE GROWTH FACTOR-I AEROSOL FORMULATIONS FOR PULMONARY DELIVERY CHAPTER III ........................................................................... 73 PULMONARY INSULIN-LIKE GROWTH FACTOR I DELIVERY FROM TREHALOSE AND SILK-FIBROIN MICROPARTICLES CHAPTER IV ......................................................................... 113 EXPRESSION OF IGF-I MUTANTS CONCLUSION AND OUTLOOK ......................................... 147 DOCUMENTATION OF AUTHORSHIP ............................. 159 CURRICULUM VITAE ......................................................... 163 ACKNOWLEDGMENTS ..................................................... -
Mecasermin in Insulin Receptor-Related Severe Insulin Resistance Syndromes: Case Report and Review of the Literature
International Journal of Molecular Sciences Review Mecasermin in Insulin Receptor-Related Severe Insulin Resistance Syndromes: Case Report and Review of the Literature Michaela Plamper, Bettina Gohlke, Felix Schreiner and Joachim Woelfle * Pediatric Endocrinology and Diabetology Division, Children’s Hospital, University of Bonn, Adenauerallee 119, 53113 Bonn, Germany; [email protected] (M.P.); [email protected] (B.G.); [email protected] (F.S.) * Correspondence: Joachim.Woelfl[email protected]; Tel.: +49-228-2873-3223; Fax: +49-228-2873-3472 Received: 8 March 2018; Accepted: 18 April 2018; Published: 24 April 2018 Abstract: Mutations in the insulin receptor (INSR) gene underlie rare severe INSR-related insulin resistance syndromes (SIR), including insulin resistance type A, Rabson–Mendenhall syndrome and Donohue syndrome (DS), with DS representing the most severe form of insulin resistance. Treatment of these cases is challenging, with the majority of DS patients dying within the first two years of life. rhIGF-I (mecasermin) has been reported to improve metabolic control and increase lifespan in DS patients. A case report and literature review were completed. We present a case involving a male patient with DS, harbouring a homozygous mutation in the INSR gene (c.591delC). Initial rhIGF-I application via BID (twice daily) injection was unsatisfactory, but continuous subcutaneous rhIGF-I infusion via an insulin pump improved weight development and diabetes control (HbA1c decreased from 10 to 7.6%). However, our patient died at 22 months of age during the course of a respiratory infection in in Libya. Currently available data in the literature comprising more than 30 treated patients worldwide seem to support a trial of rhIGF-I in SIR. -
The Use of Stems in the Selection of International Nonproprietary Names (INN) for Pharmaceutical Substances
WHO/PSM/QSM/2006.3 The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances 2006 Programme on International Nonproprietary Names (INN) Quality Assurance and Safety: Medicines Medicines Policy and Standards The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances FORMER DOCUMENT NUMBER: WHO/PHARM S/NOM 15 © World Health Organization 2006 All rights reserved. Publications of the World Health Organization can be obtained 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 noncommercial distribution – should be addressed to WHO Press, at the above address (fax: +41 22 791 4806; e-mail: [email protected]). 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 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. Errors and omissions excepted, the names of proprietary products are distinguished by initial capital letters. -
Copyright by Daniel Lukas Rosenke 2020 the Dissertation Committee for Daniel Lukas Rosenke Certifies That This Is the Approved Version of the Following Dissertation
Copyright by Daniel Lukas Rosenke 2020 The Dissertation Committee for Daniel Lukas Rosenke Certifies that this is the approved version of the following Dissertation: Supply and Enhance: Tracing the Doping Supply Chain in the 1980s Committee: Janice S. Todd, Supervisor Thomas M. Hunt Tolga Ozyurtcu John Hoberman Ian Ritchie Supply and Enhance: Tracing the Doping Supply Chain in the 1980s by Daniel Lukas Rosenke Dissertation Presented to the Faculty of the Graduate School of The University of Texas at Austin in Partial Fulfilment of the Requirements for the degree of Doctor of Philosophy The University of Texas at Austin August 2020 Dedication To my mother, the strongest woman I know To Adam: my brother, best friend, and forever my partner in crime Acknowledgements This project may never have come to fruition without the influence of father, Scott Rosenke. In my young and more impressionable years, he molded me into a man of confidence and conviction, and inspired in me the unwavering self-belief to pursue my dreams, no matter how far-fetched. Perhaps most significantly, I credit him with first introducing me to the subject matter I discuss in this volume, and piquing my interest in the surreptitious drug culture in Olympic and professional sports. Sometime in our mid-teens, I recall my brother Adam – my handsome identical twin – and I seated on the couch with Dad watching Lance Armstrong’s second Tour de France victory. At the time many believed the brash cycling maverick from Plano, Texas, a folk hero among cancer survivors worldwide, was a manna from heaven, sent to restore faith in the sport after a widely-reported scandal at the Tour two years earlier. -
Mecasermin Rinfabate for the Prevention of Retinopathy of Prematurity in Neonates of Less Than 32 Weeks of Gestational Age
European Medicines Agency Pre-authorisation Evaluation of Medicines for Human Use Document Date: London, 24 April 2009 Doc.Ref.: EMEA/COMP/63851/2008 Committee for Orphan Medicinal Products Public summary of positive opinion for orphan designation of mecasermin rinfabate for the prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age On 28 August 2006, orphan designation (EU/3/06/399) was granted by the European Commission to ROP Pharma AB, Sweden, for mecasermin rinfabate for the prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age. The sponsor changed name to Premacure AB in September 2007. What is the retinopathy of prematurity? Children that are born preterm are deprived of their natural environment in the womb, and they lack important factors normally provided to the unborn child, such as proteins, cellular growth factors and cytokines. The last weeks of a full-term pregnancy are also important for the growth of the eye, in particular for the formation of blood vessels supplying blood to the retina (the area at the back of the eye that receives light and sends pictures of what the eye sees to the brain). In some premature infants, the normal growth of the retinal vessels stops, and abnormal new vessels begin to grow. Therefore, the oxygen supply to the retina is limited. The formation of abnormal vessels is accompanied by the production of scar tissue. In some cases this can result in visual impairment and in extreme cases in blindness. What is the estimated number of patients at risk of developing the condition? At the time of designation the population at risk of developing retinopathy of prematurity in neonates of less than 32 weeks of gestational age was approximately 1.2 in 10,000 people in the European Union (EU) *. -
Note Balancing the Justices in Anti
Note Balancing the Justices in Anti-Doping Law: The Need to Ensure Fair Athletic Competition Through Effective Anti-Doping Programs vs. the Protection of Rights of Accused Athletes Ryan Connolly∗ Introduction................................................................................................... 42 I. Structure of the Current International System .......................................... 43 A. Court of Arbitration for Sport........................................................... 43 B. U.S. Anti-Doping Agency ................................................................ 45 II. Unique Issues Contributing to Anti-Doping’s Jurisprudential Community ............................................................................................ 46 A. Scientific Limitations........................................................................ 46 B. Pharmaceutical Innovations.............................................................. 49 C. Dietary Supplements......................................................................... 50 D. Designer Steroids.............................................................................. 52 E. Effect on Anti-Doping Law.............................................................. 54 III. Anti-Doping Jurisprudence ..................................................................... 54 A. Procedural Issues.............................................................................. 54 ∗ The author of this essay has been the Business Manager and Consultant for the UCLA Olympic Analytical -
The Tim Ferriss Show Transcripts Episode 143: Patrick Arnold Show Notes and Links at Tim.Blog/Podcast
The Tim Ferriss Show Transcripts Episode 143: Patrick Arnold Show notes and links at tim.blog/podcast Tim Ferriss: Greetings, my little Mogwai. This is Tim Ferriss, and I'm going to redo a legal disclaimer for this episode. I don't do many of these, but at the behest of my ever-so-competent legal counsel, allow me to read the following. I am not recommending, endorsing or supporting any of the substances or compounds discussed or described in this interview, particularly, when this applies to illegal, illicit or dangerous compounds or substances. I am interviewing Patrick, furthermore, as a journalist seeking additional information regarding matters of public interest and concern. You should also notice that we have removed a number of names of different folks to protect the innocent, guilty or otherwise. With all of that said, since I've covered my little white ass, please enjoy. [Intro] Hello you freaks out there, and aren't we all freaks at the end of the day? This is Tim Ferriss, and welcome to another episode of The Tim Ferriss Show. If you liked my episode with Dom D’Agostino, the incredible scientist who fasted six, seven days, whatever it was, dead-lifted 500 pounds for ten reps, and continues to do amazing work for the Department of Defense with exogenous ketones, etc., etc., you might love this one. So here we go. Patrick Arnold is widely considered the father of prohormones. He's also an organic chemist known for introducing androstenedione – remember Mark McGuire – 1 androstenediol, marketed as 1AD, and methylhexanamine, if I'm getting that correct, into the dietary supplement market. -
A Behavioral Law and Economics Approach to Legal Regulation of Doping in Sports Shayna M
Marquette Sports Law Review Volume 19 Article 7 Issue 1 Fall Are We All Dopes? A Behavioral Law and Economics Approach to Legal Regulation of Doping in Sports Shayna M. Sigman Follow this and additional works at: http://scholarship.law.marquette.edu/sportslaw Part of the Entertainment and Sports Law Commons Repository Citation Shayna M. Sigman, Are We All Dopes? A Behavioral Law and Economics Approach to Legal Regulation of Doping in Sports, 19 Marq. Sports L. Rev. 125 (2008) Available at: http://scholarship.law.marquette.edu/sportslaw/vol19/iss1/7 This Symposium is brought to you for free and open access by the Journals at Marquette Law Scholarly Commons. For more information, please contact [email protected]. ARE WE ALL DOPES? A BEHAVIORAL LAW & ECONOMICS APPROACH TO LEGAL REGULATION OF DOPING IN SPORTS SHAYNA M. SIGMAN* INTRODUCTION You should care about the prevalence of performance-enhancing drugs (PEDs) and doping methods currently being used and abused within elite sports. You should care, not because teenagers and youth are harmed by this, even though they most likely are. 1 You should care, not because elite athletes or those attempting to become elite athletes are harmed by this, even though they most likely are. 2 Whether you are a recreational athlete or a couch potato, whether you are a serious sports fan, a casual fan, or not a fan at all, you should care about the fact that it is highly likely that you have been harmed by doping in sports. Associate Professor of Law, Jacob D. Fuchsberg Touro Law Center. -
(Mecasermin Rinfabate [Rdna Origin] Injection) DESCRIPTION IPLEX
PACKAGE INSERT IPLEX™ (mecasermin rinfabate [rDNA origin] injection) DESCRIPTION IPLEX™ (mecasermin rinfabate [rDNA origin] injection) is an aqueous solution for injection containing a binary protein complex of human insulin-like growth factor-1 (rhIGF-1) and human insulin-like growth factor-binding protein-3 (rhIGFBP-3), both produced by recombinant DNA technology. rhIGF-1 and rhIGFBP-3 are produced by two separate E. coli strains: one containing the human gene for insulin-like growth factor-1 (IGF-1), the other containing the human gene for insulin-like growth factor-binding protein-3 (IGFBP-3). IGF-1 consists of 70 amino acids in a single chain with three intramolecular disulfide bridges and a molecular weight of 7649 daltons. The amino acid sequence of the rhIGF-1 protein is identical to that of endogenous human IGF-1. IGFBP-3 consists of 264 amino acid residues with a molecular weight of 28,732 daltons. The amino acid sequence of the rhIGFBP-3 protein is identical to that of endogenous human IGFBP-3. Endogenous IGFBP-3 contains 18 cysteine residues that are all paired in disulfide bonds to form the biologically active molecule, but the pairings have not been fully elucidated. The rhIGF-1 and rhIGFBP-3 proteins are complexed in a 1:1 molar ratio for formation of mecasermin rinfabate with a molecular weight of 36,381 daltons. IGFBP-3 from human plasma is glycosylated, whereas rhIGFBP-3 produced in E. coli is non-glycosylated. Glycosylated and non- glycosylated IGFBP-3 bind IGF-1 with similar affinities. Primary structures for rhIGF-1/rhIGFBP-3: -
Insulin-Like Growth Factor Binding Proteins: a Structural Perspective, Frontiers in Endocrinology, 2012; 3:38
PUBLISHED VERSION Forbes, Briony Evelyn; McCarthy, Peter James; Norton, R. Insulin-like growth factor binding proteins: a structural perspective, Frontiers in Endocrinology, 2012; 3:38 © 2012 Forbes, McCarthy and Norton. This is an open-access article distributed under the terms of the Creative Commons Attribution Non Commercial License, which permits non-commercial use, distribution, and reproduction in other forums, provided the original authors and source are credited. Originally published by Frontiers Research Foundation – http://www.frontiersin.org/Molecular_and_Structural_Endocrinology/10.3389/fendo.2012.00038/abstract PERMISSIONS http://www.frontiersin.org/Copyright.aspx The licence granted to third parties is a Creative Common Attribution ("CC BY") licence. http://www.frontiersin.org/WebTerms.aspx Subject to the other provisions of these conditions and unless otherwise indicated, - The CC BY licence granted to you above means that, except as stated in any Rules and/or Third- Party Rules, and subject to your complying strictly with any copyright notices, you may download, print, re-publish (but not in a Non-Acceptable Forum) and reuse and republish Journal Articles published on Frontiers Websites, subject to your obligation to acknowledge prominently that such materials were first published by Frontiers and to acknowledge prominently (in accordance with established academic practice) the identity of the author or authors, and your obligation to maintain, reproduce and comply with all copyright and other intellectual property rights notices and signs. 6th December 2012 http://hdl.handle.net/2440/72602 REVIEW ARTICLE published: 02 March 2012 doi: 10.3389/fendo.2012.00038 Insulin-like growth factor binding proteins: a structural perspective Briony E. -
(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.