Cover Image
市場調查報告書

龐貝氏症:開發中產品分析

Pompe Disease - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 200991
出版日期 內容資訊 英文 70 Pages
訂單完成後即時交付
價格
Back to Top
龐貝氏症:開發中產品分析 Pompe Disease - Pipeline Review, H2 2016
出版日期: 2016年10月29日 內容資訊: 英文 70 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龐貝氏症治療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

治療藥的開發

  • 開發中產品的概要
  • 開發中產品比較分析

龐貝氏症:企業開發中的治療藥

龐貝氏症:大學/機關研究中的治療藥

龐貝氏症:開發中產品概況

  • 後期階段的產品
  • 臨床階段的產品
  • 初期階段的產品

龐貝氏症:企業開發中的產品

龐貝氏症:大學/機關研究中的產品

龐貝氏症的治療藥開發企業

  • Alexion Pharmaceuticals, Inc.
  • Amicus Therapeutics, Inc.
  • Audentes Therapeutics, Inc.
  • BioMarin Pharmaceutical Inc.
  • EpiVax, Inc.
  • Etubics Corporation
  • Genzyme Corporation
  • Greenovation Biotech GmbH
  • Oxyrane Belgium NV
  • Pharming Group N.V.
  • RespireRx Pharmaceuticals Inc.
  • Sarepta Therapeutics, Inc.

龐貝氏症:治療藥的評估

  • 單劑產品
  • 組合產品
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

龐貝氏症:最近的開發平台趨勢

龐貝氏症:暫停中的計劃

龐貝氏症:產品開發的里程碑

  • 主要消息和新聞稿

附錄

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC8619IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease - Pipeline Review, H2 2016, 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, H2 2016, 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, Phase I, Preclinical and Discovery stages are 1, 1, 2, 8 and 2 respectively for Similarly, the Universities portfolio in Phase II and Phase I stages comprises 1 and 1 molecules, respectively for Pompe Disease.

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
  • Therapeutics Development
    • Pipeline Products for Pompe Disease - Overview
    • Pipeline Products for Pompe Disease - Comparative Analysis
  • Pompe Disease - Therapeutics under Development by Companies
  • Pompe Disease - Therapeutics under Investigation by Universities/Institutes
  • Pompe Disease - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
  • Pompe Disease - Products under Development by Companies
  • Pompe Disease - Products under Investigation by Universities/Institutes
  • Pompe Disease - Companies Involved in Therapeutics Development
    • Alexion Pharmaceuticals Inc
    • Amicus Therapeutics, Inc.
    • Audentes Therapeutics, Inc.
    • EpiVax, Inc.
    • Etubics Corporation
    • Genzyme Corporation
    • greenovation Biotech GmbH
    • Oxyrane Belgium NV
    • Pharming Group N.V.
    • RespireRx Pharmaceuticals Inc.
    • Sarepta Therapeutics, Inc.
  • Pompe Disease - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Combination Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • 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
    • CX-1739 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CX-717 - 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
    • GZ-402666 - 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
    • Recombinant Alpha Glucosidase Replacement for Pompe Disease - 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
    • Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - 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, 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
      • May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders
      • May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Pompe Disease, H2 2016
  • Number of Products under Development for Pompe Disease - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Products under Investigation by Universities/Institutes, H2 2016
  • Pompe Disease - Pipeline by Alexion Pharmaceuticals Inc, H2 2016
  • Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H2 2016
  • Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H2 2016
  • Pompe Disease - Pipeline by EpiVax, Inc., H2 2016
  • Pompe Disease - Pipeline by Etubics Corporation, H2 2016
  • Pompe Disease - Pipeline by Genzyme Corporation, H2 2016
  • Pompe Disease - Pipeline by greenovation Biotech GmbH, H2 2016
  • Pompe Disease - Pipeline by Oxyrane Belgium NV, H2 2016
  • Pompe Disease - Pipeline by Pharming Group N.V., H2 2016
  • Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc., H2 2016
  • Pompe Disease - Pipeline by Sarepta Therapeutics, Inc., H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Assessment by Combination Products, H2 2016
  • Number of Products by Stage and Target, H2 2016
  • Number of Products by Stage and Mechanism of Action, H2 2016
  • Number of Products by Stage and Route of Administration, H2 2016
  • Number of Products by Stage and Molecule Type, H2 2016
  • Pompe Disease - Dormant Projects, H2 2016
  • Pompe Disease - Discontinued Products, H2 2016

List of Figures

  • Number of Products under Development for Pompe Disease, H2 2016
  • Number of Products under Development for Pompe Disease - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Targets, H2 2016
  • Number of Products by Stage and Targets, H2 2016
  • Number of Products by Mechanism of Actions, H2 2016
  • Number of Products by Stage and Mechanism of Actions, H2 2016
  • Number of Products by Routes of Administration, H2 2016
  • Number of Products by Stage and Routes of Administration, H2 2016
  • Number of Products by Molecule Types, H2 2016
  • Number of Products by Stage and Molecule Types, H2 2016
Back to Top