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市場調查報告書

龐貝氏症:開發中產品分析

Pompe Disease - Pipeline Review, H1 2017

出版商 Global Markets Direct 商品編碼 200991
出版日期 內容資訊 英文 52 Pages
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龐貝氏症:開發中產品分析 Pompe Disease - Pipeline Review, H1 2017
出版日期: 2017年05月16日 內容資訊: 英文 52 Pages
簡介

龐貝氏症(第Ⅱ型糖原病)是遺傳性疾病之一,初發症狀為步行·呼吸用肌肉階段性衰弱。特別是幼兒發病時,往往對心肌造成嚴重的影響。酸性麥芽糖酵素這種具有肝醣分解功能的酵素突變,是引起該疾病的原因。發病的結果會造成肝醣在體內(尤其是肌肉)裡堆蓄,而打擊到各個細胞。

本報告提供全球各國治療龐貝氏症所用之開發中產品開發情形相關分析,提供您開發中產品的最新趨勢,及臨床實驗的各階段產品一覽,主要企業簡介,主要藥物概要,最新的產業趨勢等資訊。

簡介

  • 調查範圍

龐貝氏症 - 概要

龐貝氏症 - 治療藥的開發

  • 開發平台概要
  • 各企業的開發平台趨勢
  • 各大學·研究機關的開發平台趨勢
  • 企業開發中的產品
  • 大學·研究機關開發中的產品

龐貝氏症 - 治療藥的評估

  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

龐貝氏症的開發治療藥的企業

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • EpiVax Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Huons Co Ltd
  • JCR Pharmaceuticals Co Ltd
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Sarepta Therapeutics Inc

藥物簡介

龐貝氏症 - 暫停中的計劃

龐貝氏症 - 開發中止的產品

龐貝氏症 - 產品開發的里程碑

  • 主要消息和新聞稿

附錄

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目錄
Product Code: GMDHC9265IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease - Pipeline Review, H1 2017, provides an overview of the Pompe Disease (Metabolic Disorders) pipeline landscape.

Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache and difficulty chewing or jaw muscle fatigue.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Pompe Disease (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Pompe Disease (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Pompe Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 8 and 2 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively.

Pompe Disease (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Pompe Disease (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Pompe Disease (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Pompe Disease (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Pompe Disease (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Pompe Disease (Metabolic Disorders)

Reasons to buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Pompe Disease (Metabolic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Pompe Disease (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Pompe Disease - Overview
    • Pompe Disease - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Pompe Disease - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Pompe Disease - Companies Involved in Therapeutics Development
    • Amicus Therapeutics Inc
    • Audentes Therapeutics Inc
    • EpiVax Inc
    • Etubics Corp
    • Genzyme Corp
    • greenovation Biotech GmbH
    • Huons Co Ltd
    • JCR Pharmaceuticals Co Ltd
    • Oxyrane Belgium NV
    • Pharming Group NV
    • Sarepta Therapeutics Inc
  • Pompe Disease - Drug Profiles
    • Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AT-982 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ATB-200 + miglustat - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • GZ-402666 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • HU-023 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • JR-162 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MOSS-GAA - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • OXY-2810 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PGN-004 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Synthetic Peptides for Pompe disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VAL-1221 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Pompe Disease - Dormant Projects
  • Pompe Disease - Discontinued Products
  • Pompe Disease - Product Development Milestones
    • Featured News & Press Releases
      • Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease
      • Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK
      • Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
      • Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease
      • Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium
      • May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Pompe Disease, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Products under Development by Companies, H1 2017
  • Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Stage and Target, H1 2017
  • Number of Products by Stage and Mechanism of Action, H1 2017
  • Number of Products by Stage and Route of Administration, H1 2017
  • Number of Products by Stage and Molecule Type, H1 2017
  • Pompe Disease - Pipeline by Amicus Therapeutics Inc, H1 2017
  • Pompe Disease - Pipeline by Audentes Therapeutics Inc, H1 2017
  • Pompe Disease - Pipeline by EpiVax Inc, H1 2017
  • Pompe Disease - Pipeline by Etubics Corp, H1 2017
  • Pompe Disease - Pipeline by Genzyme Corp, H1 2017
  • Pompe Disease - Pipeline by greenovation Biotech GmbH, H1 2017
  • Pompe Disease - Pipeline by Huons Co Ltd, H1 2017
  • Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017
  • Pompe Disease - Pipeline by Oxyrane Belgium NV, H1 2017
  • Pompe Disease - Pipeline by Pharming Group NV, H1 2017
  • Pompe Disease - Pipeline by Sarepta Therapeutics Inc, H1 2017
  • Pompe Disease - Dormant Projects, H1 2017
  • Pompe Disease - Discontinued Products, H1 2017

List of Figures

  • Number of Products under Development for Pompe Disease, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Stage and Top 10 Targets, H1 2017
  • Number of Products by Mechanism of Actions, H1 2017
  • Number of Products by Stage and Mechanism of Actions, H1 2017
  • Number of Products by Routes of Administration, H1 2017
  • Number of Products by Stage and Routes of Administration, H1 2017
  • Number of Products by Molecule Types, H1 2017
  • Number of Products by Stage and Molecule Types, H1 2017
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