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市場調查報告書

龐貝氏症的全球市場 (2015∼2019年)

Global Pompe Disease Market 2015-2019

出版商 TechNavio (Infiniti Research Ltd.) 商品編碼 328068
出版日期 內容資訊 英文 71 Pages
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龐貝氏症的全球市場 (2015∼2019年) Global Pompe Disease Market 2015-2019
出版日期: 2015年04月08日 內容資訊: 英文 71 Pages
簡介

龐貝氏症為溶小體儲積症(LSD) 之一,分為嬰兒型和成人型2種。至今有發現300種以上的遺傳基因變異為龐貝氏症的病原。在男女間和人種間發病率沒有差異。治療藥現在還沒完成,不過,飲食療法和酵素補充療法等,有效減輕臨床症狀。全球龐貝氏症市場2014∼2019年預計以11.79%的年複合成長率(CAGR) 成長。

本報告提供全球的龐貝氏症相關市場相關分析、疾病、治療藥概要和基本的市場結構、開發平台候補藥現況、市場規模趨勢 (今後5年的預測值)、各地區趨勢、推動、阻礙市場要素與其影響力、主要企業簡介、今後的市場機會等調查、考察。

第1章 摘要整理

第2章 簡稱清單

第3章 調查範圍

  • 市場概要
  • 主要的產品

第4章 市場調查手法

  • 市場調查流程
  • 調查手法

第5章 簡介

第6章 疾病概要

  • 關於疾病
    • 龐貝氏症的種類
    • 症狀
    • 診斷
  • 病因及病原
    • 生物化學的途徑
  • 流行病學
  • 管理
  • 經濟負擔

第7章 市場形勢

  • 市場概要
  • 市場規模與預測
  • 波特的五力分析

第8章 開發平台分析

  • 開發平台候補相關主要資訊
    • rAAV1-CMV-GaAs(砷化鎵)
    • OXY-2810
    • 龐貝氏症用Tregitopes
    • AT-002
    • LSD用ZFP轉錄因子
    • VAL-1221

第9章 地區區分

第10章 購買標準

第11章 推動市場成長要素

第12章 成長推動因素與其影響

第13章 市場課題

第14章 成長推動因素與課題的影響

第15章 市場趨勢

第16章 趨勢與其影響

第17章 業者情勢

  • 競爭模式
    • 主要消息
    • 企業合併、收購 (M&A)
  • 市場佔有率分析
  • 其他的供應商,及未來的有力供應商

第18章 主要供應商分析

  • Amicus Therapeutics
    • 主要資料
    • 產業概要
    • 產業策略
    • 主要的開發
    • SWOT分析
  • BioMarin Pharmaceutical
  • Genzyme

第19章 相關報告

圖表一覽

目錄
Product Code: IRTNTR5625

About Pompe Disease

Pompe disease is a type of LSD. LSD refers to a group of diseases arising from the accumulation of specific substrates. LSDs are progressive and have variable age of onset and clinical symptoms. LSDs are inherited autosomal recessive disorders. Pompe disease has two broad sub-types: infantile-onset Pompe disease and adult-onset Pompe disease. Pompe disease occurs as a result of mutations in the GAA gene. These mutations are responsible for the preventing the action of the enzyme acid alpha-glucosidase, which is responsible for catabolizing glycogen, thereby allowing sugar to accumulate and reach toxic levels in the lysosomes of cells. The buildup of glycogen in certain organs and tissues, mostly in the muscles, hinders the normal functions of organs. Pompe disease is an autosomal recessive disease. This implies that both the parents pass on one abnormally mutated copy of the gene to their child. A parent carrying an abnormal copy of the gene is known as a carrier. Pompe disease affects both men and women, irrespective of the gender. Pompe disease affects people across all ethnicities and races. Individuals affected with Pompe disease acquire the disease at birth, however, the onset of symptoms varies extensively. More than 300 genetic mutations that can cause Pompe disease have been identified by the researchers till date. Though there is no cure for Pompe disease, diet therapy and enzyme replacement therapy are effective in reducing the clinical manifestations of the disease.

TechNavio's analysts forecast the Global Pompe Disease market to grow at a CAGR of 11.79 percent over the period 2014-2019.

Covered in this Report

This report covers the present scenario and the growth prospects of the Global Pompe Disease market for the period 2015-2019. To calculate the market size, the report considers revenue from the sales of drugs available in the market for the management of Pompe disease.

The report also presents the vendor landscape and a corresponding detailed analysis of the major vendors in the Global Pompe Disease market. The report also considers the revenue that is likely to be generated from the drug candidates that are under various phases of development. In addition, it discusses the major drivers that influence the growth of the market, the challenges faced by the vendors and the market at large, as well as the key trends that contribute to the growth the market.

TechNavio's report, Global Pompe Disease Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, and the EMEA and APAC regions; it also covers the Global Pompe Disease market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

Key Regions

  • Americas
  • APAC
  • EMEA

Key Vendors

  • Amicus Therapeutics
  • BioMarin Pharmaceutical
  • Genzyme

Other Prominent Vendors

  • Audentes Therapeutics
  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Valerion Therapeutics

Market Driver

  • Special Provisions for Orphan Drugs
  • For a full, detailed list, view our report

Market Challenge

  • Limited Patient Population
  • For a full, detailed list, view our report

Market Trend

  • Regulatory Assistance in Emerging Nations
  • For a full, detailed list, view our report

Key Questions Answered in this Report

  • What will the market size be in 2019 and what will the growth rate be?
  • What are the key market trends?
  • What is driving this market?
  • What are the challenges to market growth?
  • Who are the key vendors in this market space?
  • What are the market opportunities and threats faced by the key vendors?
  • What are the strengths and weaknesses of the key vendors?

Table of Contents

01. Executive Summary

02. List of Abbreviations

03. Scope of the Report

  • 03.1. Market Overview
  • 03.2. Product Offerings

04. Market Research Methodology

  • 04.1. Market Research Process
  • 04.2. Research Methodology

05. Introduction

06. Disease Overview

  • 06.1. Understanding the Disease
    • 06.1.1. Types of Pompe Disease
    • 06.1.2. Symptoms
    • 06.1.3. Diagnosis
  • 06.2. Etiology and Pathogenesis
    • 06.2.1. Biochemical Pathway
  • 06.3. Epidemiology
  • 06.4. Management
  • 06.5. Economic Burden

07. Market Landscape

  • 07.1. Market Overview
  • 07.2. Market Size and Forecast
  • 07.3. Five Forces Analysis

08. Pipeline Analysis

  • 08.1. Key Information on Pipeline Candidates
    • 08.1.1. rAAV1-CMV-GAA
    • 08.1.2. OXY-2810
    • 08.1.3. Tregitopes for Pompe Disease
    • 08.1.4. AT-002
    • 08.1.5. ZFP Transcription Factors for LSDs
    • 08.1.6. VAL-1221

09. Geographical Segmentation

10. Buying Criteria

11. Market Growth Drivers

12. Drivers and their Impact

13. Market Challenges

14. Impact of Drivers and Challenges

15. Market Trends

16. Trends and their Impact

17. Vendor Landscape

  • 17.1. Competitive Scenario
    • 17.1.1. Key News
    • 17.1.2. Mergers and Acquisitions
  • 17.2. Market Share Analysis 2014
    • 17.2.1. Genzyme
    • 17.2.2. BioMarin Pharmaceutical
    • 17.2.3. Amicus Therapeutics
  • 17.3. Other and Future Prominent Vendors

18. Key Vendor Analysis

  • 18.1. Amicus Therapeutics
    • 18.1.1. Key Facts
    • 18.1.2. Business Overview
    • 18.1.3. Business Strategy
    • 18.1.4. Key Developments
    • 18.1.5. SWOT Analysis
  • 18.2. BioMarin Pharmaceutical
    • 18.2.1. Key Facts
    • 18.2.2. Business Overview
    • 18.2.3. Segmentation by Revenue 2014
    • 18.2.4. Segmentation by Revenue 2013 and 2014
    • 18.2.5. Geographical Segmentation by Revenue 2014
    • 18.2.6. Business Strategy
    • 18.2.7. Recent Developments
    • 18.2.8. SWOT Analysis
  • 18.3. Genzyme
    • 18.3.1. Key Facts
    • 18.3.2. Business Overview
    • 18.3.3. Product Segmentation by Revenue 2013
    • 18.3.4. Product Segmentation by Revenue 2012 and 2013
    • 18.3.5. Geographical Segmentation
    • 18.3.6. Business Strategy
    • 18.3.7. Recent Developments
    • 18.3.8. SWOT Analysis

19. Other Reports in this Series

List of Exhibits:

  • Exhibit 1: Market Research Methodology
  • Exhibit 2: Snapshot of Pompe Disease
  • Exhibit 3: Heredogram of Pompe Disease
  • Exhibit 4: Types of Pompe Disease
  • Exhibit 5: Signs and Symptoms of Pompe Disease
  • Exhibit 6: Differential Diagnosis of Pompe Disease in Infants
  • Exhibit 7: Differential Diagnosis of Pompe Disease in Children and Adults
  • Exhibit 8: Biochemical Pathway of Pompe Disease
  • Exhibit 9: Approaches to Treat Pompe Disease
  • Exhibit 10: Economic Burden of Pompe Disease
  • Exhibit 11: Snapshot of Global Pompe Disease Market 2014
  • Exhibit 12: Global Pompe Disease Market 2014-2019 (US$ million)
  • Exhibit 13: Drivers and Challenges of Global Pompe Disease Market
  • Exhibit 14: Pipeline Portfolio of Global Pompe Disease Market
  • Exhibit 15: Segmentation of Global Pompe Disease Market by Geography 2014
  • Exhibit 16: Genzyme: Product Portfolio
  • Exhibit 17: BioMarin Pharmaceutical: Segmentation by Revenue 2014
  • Exhibit 18: BioMarin Pharmaceutical: Segmentation by Revenue 2013 and 2014 (US$ million)
  • Exhibit 19: BioMarin Pharmaceutical: Geographical Segmentation by Revenue 2014
  • Exhibit 20: Genzyme: Product Segmentation by Revenue 2013
  • Exhibit 21: Genzyme: Product Segmentation by Revenue 2012 and 2013 (US$ million)
  • Exhibit 22: Genzyme: Geographical Segmentation
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