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GH/IGF-1 Abnormalities and Muscle Impairment: from Basic Research to Clinical Practice
International Journal of Molecular Sciences Review GH/IGF-1 Abnormalities and Muscle Impairment: From Basic Research to Clinical Practice Betina Biagetti * and Rafael Simó * Diabetes and Metabolism Research Unit, Vall d’Hebron Research Institute and CIBERDEM (ISCIII), Universidad Autónoma de Barcelona, 08193 Bellaterra, Spain * Correspondence: [email protected] (B.B.); [email protected] (R.S.); Tel.: +34-934894172 (B.B.); +34-934894172 (R.S.) Abstract: The impairment of skeletal muscle function is one of the most debilitating least understood co-morbidity that accompanies acromegaly (ACRO). Despite being one of the major determinants of these patients’ poor quality of life, there is limited evidence related to the underlying mechanisms and treatment options. Although growth hormone (GH) and insulin-like growth factor-1 (IGF-1) levels are associated, albeit not indisputable, with the presence and severity of ACRO myopathies the precise effects attributed to increased GH or IGF-1 levels are still unclear. Yet, cell lines and animal models can help us bridge these gaps. This review aims to describe the evidence regarding the role of GH and IGF-1 in muscle anabolism, from the basic to the clinical setting with special emphasis on ACRO. We also pinpoint future perspectives and research lines that should be considered for improving our knowledge in the field. Keywords: acromegaly; myopathy; review; growth hormone; IGF-1 1. Introduction Acromegaly (ACRO) is a rare chronic disfiguring and multisystem disease due to Citation: Biagetti, B.; Simó, R. non-suppressible growth hormone (GH) over-secretion, commonly caused by a pituitary GH/IGF-1 Abnormalities and Muscle tumour [1]. -
Growth Hormone Booklet
GROWTH HORMONE AND PRADER-WILLISECOND EDITION SYNDROME A REFerence For FaMILies and Care ProViders Donald G. Goranson, Jr., Editor Growth Hormone and Prader-Willi Syndrome — Second Edition Published By: Prader-Willi Syndrome Association (Usa) 8588 Potter Park Drive, Suite 500 Sarasota, Florida 34238 Toll Free: 800-926-4797 Local: 941-312-0400 Fax: 941-312-0142 www.pwsausa.org © Copyright 2011 A Reference for Families and Care Providers Growth HORMONE AND PraderSECOND-WILLI EDITION Syndrome A REFerence For FaMILies and Care ProViders Donald G. Goranson, Jr., Editor Growth Hormone and Prader-Willi Syndrome — Second Edition A Reference for Families and Care Providers CONTENTS Acknowledgments....................................................................... 5 Introduction and History .............................................................. 7 Prader-Willi Syndrome and Growth ...................................................... 9 Effects of Growth Hormone Treatment in Children with PWS ................................ 20 What Is Involved in Growth Hormone Treatment? .......................................... 26 Questions, Wisdom and Survey Data from our Families ..................................... 34 Appendix: A. Overview of Prader-Willi Syndrome................................................ 40 B. Information Resources on Prader-Willi Syndrome .................................... 42 C. Information Resources on Growth Hormone Use and Products ........................ 43 D. Glossary of Terms ............................................................ -
Fully Human Domain Antibody Therapeutics: the Best of Both Worlds
Drug Discovery Fully Human Domain Antibody Therapeutics: The Best of Both Worlds By combining the therapeutic benefits of small molecule drugs with those of fully human antibodies, Domain Antibodies are expected to have strong therapeutic and commercial potential. By Robert Connelly at Domantis Robert Connelly is Chief Executive Officer of Domantis. He has over 22 years’ commercial experience of the life science sector, including that gained in the fields of diagnostics, drug discovery technologies and antibody therapeutics. Prior to joining Domantis, he was CEO of Veritas Pharmaceuticals (Los Angeles, USA), an in vivo imaging start-up company. He spent over five years with IGEN International, latterly as Senior Vice President and General Manager, Life Sciences, where he took part in the company’s IPO and financing rounds, raising $130 million. The first 11 years of his career were spent at Abbott Laboratories in sales, marketing and management positions. Domain Antibodies (dAbs) are the smallest functional variable regions of either the heavy (VH) or light (VL) binding units of antibodies. At Domantis, we are chains of human antibodies. Domantis scientists applying our proprietary know-how in dAbs to deliver have used the variable domains sequences of human human therapies that address large, unmet medical antibodies to create a series of large and highly needs in areas such as inflammation, cancer and functional libraries of fully human dAbs, with each autoimmune diseases. Three and a half years after library comprising at least 1010 different dAbs. The opening our laboratories, we have a dozen proprietary dAbs selected from these libraries are both specific therapeutic programmes underway, and an additional for their biological target and are well folded and eight therapeutic programmes with partners. -
Moderna Appoints Oncology Leader Dr. Stephen Kelsey As President of Onkaido
Moderna Appoints Oncology Leader Dr. Stephen Kelsey as President of Onkaido Former Genentech executive to lead Moderna’s first venture company, focused exclusively in novel biology for oncology drug development CAMBRIDGE, Mass., July 1, 2014— Moderna Therapeutics, the pioneer in developing messenger RNA (mRNA) TherapeuticsTM, a revolutionary treatment modality to enable the in vivo production of therapeutic proteins, announced today that Stephen Kelsey, M.D., will become president of Onkaido Therapeutics, Moderna’s oncology drug development company, effective July 21. Launched in January of this year, Onkaido is Moderna’s first venture company, focused exclusively on developing and commercializing mRNA-based oncology treatments. “As we continue to grow Moderna and perfect our mRNA Therapeutics platform, we are also focused on building a transformational oncology company that will benefit patients and society. This requires hiring the best oncology talent to lead Onkaido,” said Stéphane Bancel, president and founding chief executive officer, Moderna. “Steve brings a wealth of experience in oncology drug development to his new role. His knowledge and leadership, combined with the team of Onkaido scientists and Moderna’s innovative mRNA technology, will help speed a new class of cancer drugs to patients around the world.” Dr. Kelsey has extensive pharmaceutical industry experience in oncology. After 16 years as an academic clinician, he started his industry career at Sugen, and later was vice president of hematology/oncology at Genentech. While at Genentech, Dr. Kelsey played a significant role in the development of key products Perjeta®, Kadcyla® and Erivedge®, as well as other molecules in the company’s oncology portfolio. He left Genentech in 2009 to run Geron’s oncology division, where he served for four years as executive vice president, research and development, and chief medical officer helping to develop therapeutics and vaccines to fight cancer. -
Power List 2018
APRIL 2018 # 40 Editorial Upfront In My View Sitting Down With Stop and look at how far Preparing for the EU’s new What can algae teach us Sophie Kornowski-Bonnet, the industry has come data protection regulation about medicine design? Roche Partnering 09 10 – 11 20 – 21 50 – 51 100 Power List 2018 www.themedicinemaker.com Continuous Growth Fibra-Cel® disks—3-D growth matrix for perfusion and continuous processes Suspend your disbelief: > Less susceptible to shear forces, The three-dimensional Fibra-Cel matrix clogging, and fouling entraps anchorage dependent and > Ideal for secreted product and vaccine suspension cells—for optimized growth production conditions and increased yields. > Suitable for GMP production > For use in autoclavable, sterilize-in- place or BioBLU® Single-Use Vessels www.eppendorf.com/Fibra-Cel Fibra-Cel® is a registered trademark owned by Imerys Minerals California, Inc., USA and licensed to Eppendorf, Inc., USA. Eppendorf®, the Eppendorf Brand Design and BioBLU® are registered trademarks of Eppendorf AG, Germany. All rights reserved, including graphics and images. Copyright © 2018 by Eppendorf AG. tmm_epp_ad_210x266_2018_04.indd 1 28.03.18 13:34 Online this Month The Power List The 2018 Power List, starting on page 24 of this issue, features 100 of the most inspirational professionals involved in A Scientist Walks into a Bar... format to the public”. Generally, drug development. The list was compiled it involves scientists speaking on a based on reader nominations and And gives a presentation as part variety of topics, from medicine, to feedback from a judging panel – but any of Pint of Science, a global science neuroscience, to robotics and more, in list will always be subjective. -
Growth Hormone Deficiency and Other Indications for Growth Hormone Therapy – Child and Adolescent
TUE Physician Guidelines GROWTH HORMONE DEFICIENCY AND OTHER INDICATIONS FOR GROWTH HORMONE THERAPY – CHILD AND ADOLESCENT 1. Medical Condition Growth Hormone Deficiency and other indications for growth hormone therapy (child/adolescent). 2. Diagnosis 1. Medical History Growth hormone deficiency (GHD) is a result of dysfunction of the hypothalamic-pituitary axis either at the hypothalamic or pituitary levels. The prevalence of GHD is estimated between 1:4000 and 1:10,000. GHD may be present in combination with other pituitary deficiencies, e.g. multiple pituitary hormone deficiency (MPHD) or as an isolated deficiency. Short stature, height more than 2 SD below the population mean, may represent GHD. Low birth weight, hypothyroidism, constitutional delay in growth puberty, celiac disease, inflammatory bowel disease, juvenile arthritis or other chronic systemic diseases as well as dysmorphic phenotypes such as Turner’s syndrome and genetic diagnoses such as Noonan’s syndrome and GH insensitivity syndrome must be considered when evaluating a child/adolescent for GHD. Pituitary tumors, cranial surgery or radiation, head trauma or CNS infections may also result in GHD. Idiopathic short stature (ISS) is defined as height below -2 SD score (SDS) without any concomitant condition or disease that could cause decreased growth (ISS is an acceptable indication for treatment with Growth Hormone in some but not all countries). Failure to treat children with GHD can result in significant physical, psychological and social consequences. Since not all children with GHD will require continued treatment into adulthood, the transition period is very important. The transition period can be defined as beginning in late puberty the time when near adult height has been attained, and ending with full adult maturation (6-7 years after achievement of adult height). -
Print Layout 1
TECHNICAL PROGRAM Monday, March 19 5:00 – 8:00 pm Welcome Reception Tuesday, March 20 8:00 – 10:00 Session 1: Setting the Conference Context and Drivers Chair: Geoff Slaff (Amgen) Roger Perlmutter (Amgen) Conquering the Innovation Deficit in Drug Discovery Helen Winkle (CDER, FDA) Regulatory Modernization 10:00 – 10:30 Break Vendor and poster review 10:30 – 12:30 Session 2: Rapid Cell Line Development and Improved Expression System Development Chairs: Timothy Charlebois (Wyeth) and John Joly (Genentech) Amy Shen (Genentech) Stable Antibody Production Cell-Line Development with an Improved Selection Process and Accelerated Timeline Mark Leonard (Wyeth) High-Performing Cell-Line Development within a Rapid and Integrated Platform Process Control Pranhitha Reddy (Amgen) Applying Quality-by-Design to Cell Line Development Lin Zhang (Pfizer) Development of a Fully-Integrated Automated System for High-Throughput Screening and Selection of Single Cells Expressing Monoclonal Antibodies 12:30 – 2:00 Lunch Vendor and poster review 2:00 – 4:30 Session 3: High-Throughput Bulk Process Development Chairs: Brian Kelley (Wyeth) and Jorg Thommes (Biogen Idec) Colette Ranucci (Merck) Development of a Multi-Well Plate System for High-Throughput Process Development Min Zhang (SAFC Biosciences) CHO Media Library – an Efficient Platform for Rapid Development and Optimization of Cell Culture Media Supporting High Production of Pharmaceutical Proteins in Chinese Hamster Ovary Cells Nigel Titchener-Hooker The Use of Ultra-Scale-Down Approaches to Enable Rapid Investigation -
Genentech Tocilizumab Letter of Authority June 24 2021
June 24, 2021 Hoffmann-La Roche, Ltd. C/O Genentech, Inc. Attention: Dhushy Thambipillai Regulatory Project Management 1 DNA Way, Bldg 45-1 South San Francisco, CA 94080 RE: Emergency Use Authorization 099 Dear Ms. Thambipillai: This letter is in response to Genentech, Inc.’s (Genentech) request that the Food and Drug Administration (FDA) issue an Emergency Use Authorization (EUA) for the emergency use of Actemra1 (tocilizumab) for the treatment of coronavirus disease 2019 (COVID-19) in certain hospitalized patients, as described in the Scope of Authorization (Section II) of this letter, pursuant to Section 564 of the Federal Food, Drug, and Cosmetic Act (the Act) (21 U.S.C. §360bbb-3). On February 4, 2020, pursuant to Section 564(b)(1)(C) of the Act, the Secretary of the Department of Health and Human Services (HHS) determined that there is a public health emergency that has a significant potential to affect national security or the health and security of United States citizens living abroad, and that involves the virus that causes coronavirus disease 2019 (COVID-19).2 On the basis of such determination, the Secretary of HHS on March 27, 2020, declared that circumstances exist justifying the authorization of emergency use of drugs and biological products during the COVID-19 pandemic, pursuant to Section 564 of the Act (21 3 U.S.C. 360bbb-3), subject to terms of any authorization issued under that section. Actemra is a recombinant humanized monoclonal antibody that selectively binds to both soluble and membrane-bound human IL-6 receptors (sIL-6R and mIL-6R) and subsequently inhibits IL- 6-mediated signaling through these receptors. -
List of Section 13F Securities
List of Section 13F Securities 1st Quarter FY 2004 Copyright (c) 2004 American Bankers Association. CUSIP Numbers and descriptions are used with permission by Standard & Poors CUSIP Service Bureau, a division of The McGraw-Hill Companies, Inc. All rights reserved. No redistribution without permission from Standard & Poors CUSIP Service Bureau. Standard & Poors CUSIP Service Bureau does not guarantee the accuracy or completeness of the CUSIP Numbers and standard descriptions included herein and neither the American Bankers Association nor Standard & Poor's CUSIP Service Bureau shall be responsible for any errors, omissions or damages arising out of the use of such information. U.S. Securities and Exchange Commission OFFICIAL LIST OF SECTION 13(f) SECURITIES USER INFORMATION SHEET General This list of “Section 13(f) securities” as defined by Rule 13f-1(c) [17 CFR 240.13f-1(c)] is made available to the public pursuant to Section13 (f) (3) of the Securities Exchange Act of 1934 [15 USC 78m(f) (3)]. It is made available for use in the preparation of reports filed with the Securities and Exhange Commission pursuant to Rule 13f-1 [17 CFR 240.13f-1] under Section 13(f) of the Securities Exchange Act of 1934. An updated list is published on a quarterly basis. This list is current as of March 15, 2004, and may be relied on by institutional investment managers filing Form 13F reports for the calendar quarter ending March 31, 2004. Institutional investment managers should report holdings--number of shares and fair market value--as of the last day of the calendar quarter as required by Section 13(f)(1) and Rule 13f-1 thereunder. -
Adult Growth Hormone Deficiency
A Guide for Patients • Patients should be sure to inject Nutropin at a different recommended place on their body each time to avoid tissue breakdown. A doctor or nurse should provide injection training and supervise the fi rst injection • The use of Nutropin therapy has not been studied in patients over 65 years of age. Elderly patients may be more sensitive to Nutropin therapy and may experience more side effects with Adult • Patients with Turner syndrome should be monitored closely by a doctor for ear infections and cardiovascular problems during Nutropin therapy Growth Hormone Defi ciency What are common possible side effects of Nutropin therapy? Common side effects reported in adults and children taking Nutropin therapy include injection site reactions. Additional common side effects in adults include swelling, For easily accessible answers, education, joint pain, and carpal tunnel syndrome. and support, visit Nutropin.com You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech or call 1-866-NUTROPIN (1-866-688-7674). at (888) 835-2555. How should you store Nutropin? 14 Nutropin must be kept refrigerated (36° to 46°F [2° to 8°C]). Do not freeze. NuSpin 15 pens must be used within 28 days of initial use. Throw away any unused Nutropin after 28 days of initial use. Before giving an injection, check the manufacturer’s expiration date on the pen. Do not use if it has expired. Do not inject medication if the solution is cloudy. Your healthcare team is your primary source of information about your treatment. -
In Adults with Growth Hormone Deficiency
NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE GUIDANCE EXECUTIVE (GE) Review of TA64; Human growth hormone (somatropin) in adults with growth hormone deficiency This guidance was issued in August 2003. The review date for this guidance is ‘within 6 months’ of the publication of trial data according to the last review update in May 2012. 1. Recommendation TA64 should be moved to the static list. That we consult on this proposal. 2. Original remit(s) To appraise the clinical and cost-effectiveness of human growth hormone in its licensed indications for the treatment of growth hormone deficiency in adults. 3. Current guidance 1.1 Recombinant human growth hormone (somatropin) treatment is recommended for the treatment of adults with growth hormone (GH) deficiency only if they fulfil all three of the following criteria. They have severe GH deficiency, defined as a peak GH response of less than 9 mU/litre (3 ng/ml) during an insulin tolerance test or a cross- validated GH threshold in an equivalent test. They have a perceived impairment of quality of life (QoL), as demonstrated by a reported score of at least 11 in the disease-specific 'Quality of life assessment of growth hormone deficiency in adults' (QoL- AGHDA) questionnaire. They are already receiving treatment for any other pituitary hormone deficiencies as required. 1.2 The QoL status of people who are given GH treatment should be re-assessed 9 months after the initiation of therapy (an initial 3-month period of GH dose titration, followed by a 6-month therapeutic trial period). GH treatment should be discontinued for those people who demonstrate a QoL improvement of less than 7 points in QoL-AGHDA score. -
Human Growth Hormone Page 1 of 60
Human Growth Hormone Page 1 of 60 Medical Policy Title: Human Growth Hormone Prior Authorization IS REQUIRED by the Member’s Contract Prior Authorization Form: BCBSKS reviews the Prior Authorization requests http://www.bcbsks.com/CustomerService/Forms/pdf/15-811_Growth_Hormone_PA.pdf Link to Drug List (Formulary): http://www.bcbsks.com/drugs/ Professional Institutional Original Effective Date: February 4, 1986 Original Effective Date: August 18, 2008 Revision Date(s): January 30, 2014; Revision Date(s): January 30, 2014; December 9, 2014; June 23, 2015; December 9, 2014; June 23, 2015; December 8, 2015; January 1, 2017; December 8, 2015; January 1, 2017; May 24, 2017; August 18, 2017; May 24, 2017; August 18, 2017; December 20, 2017; December 5, 2018; December 20, 2017; December 5, 2018; October 1, 2019; January 1, 2020; October 1 2019; January 1, 2020; October 1, 2020 October 1, 2020 Current Effective Date: January 1, 2020 Current Effective Date: January 1, 2020 State and Federal mandates and health plan member contract language, including specific provisions/exclusions, take precedence over Medical Policy and must be considered first in determining eligibility for coverage. To verify a member's benefits, contact Blue Cross and Blue Shield of Kansas Customer Service. The BCBSKS Medical Policies contained herein are for informational purposes and apply only to members who have health insurance through BCBSKS or who are covered by a self-insured group plan administered by BCBSKS. Medical Policy for FEP members is subject to FEP medical policy which may differ from BCBSKS Medical Policy. The medical policies do not constitute medical advice or medical care.