溶?体贮积病治疗药市场:各治疗药物，各适应症，各终端用户，各地区- 市场规模，占有率，展望，机会分析，2023年～2030年

Lysosomal Storage Diseases Therapeutics Market, By Treatment (Enzyme Replacement Therapy ), By Indication, By End User, and By Region - Size, Share, Outlook, and Opportunity Analysis, 2023 - 2030

出版日期: 2023年06月20日 | 出版商:

Coherent Market Insights | 英文 171 Pages | 商品交期: 2-3个工作天内

价格

简介目录

市场动态

溶?体贮积病的重要性日益增加、政府宣传计划的实施以及新产品的推出正在促进全球溶?体贮积病治疗市场的增长。例如，2023年5月11日，美国製药公司Chiesi USA, Inc.宣布其产品Elfabrio获得美国食品药品监督管理局批准。它在?替代疗法（ERT）中使用pegunigalsidase alfa来治疗确诊的法布里病成年患者。

本调查的主要特征

本报告提供以2021年为基准年的预测期间（2022-2030 年）之全球溶?体贮积病治疗药市场的市场规模和復合年增长率（CAGR %）相关的详细分析。
并阐明不同部门市场的潜在收入机会，说明该市场具有魅力的投资主张矩阵。
同时并提供市场推动因素和限制、机会、新产品发布或批准、市场趋势、区域前景和主要参与者采用的竞争战略等重要考察。
并基于企业亮点，各类型投资组合、主要焦点、财务业绩和市场策略等参数，介绍全球溶?体贮积病治疗药市场的主要参与者之企业简介。
藉由该报告提供的洞察，将能帮助营销人员和公司高管能够就未来的产品发布、类型升级、市场扩张和营销策略做出明智的决策。
全球牛血清市场报告配合该行业的各种利益相关者，包括投资者、供应商、产品製造商、分销商、新进入者和金融分析师。
利益相关者可以透过全球溶?体贮积病治疗药市场用于分析的各种战略矩阵来帮助他们更容易做下决策。

Lysosomal storage diseases (LSDs) are diseases caused by defects in single-genes. Enzyme defects cause nearly seventy percent of the LSDs, and the rest are defects in enzyme activator or associated proteins. A gene on a particular chromosome locus transcribes a particular enzyme, improper enzyme-coding results in inactive enzymes. Similarly, defective activators result from mutations in activator genes. These conditions cause disease of the organs in which they accumulate and decide the clinical signs and symptoms. Infants and children suffer more severely compared to adults. The clinical features are unique in many children and adults for the same disease. For example, the child's developing brain is more susceptible to insults and manifests symptoms and signs of dysfunction, while this may be milder or absent in adults. Enzyme testing is usually the initial diagnostic test, but genetic analysis of the gene mutations adds precision. There are many modern therapeutic techniques for these conditions. When applied early before organ damage sets in, these therapies have the potential to prevent or delay damage, improve quality of life, and increase lifespan.

Market Dynamics

Increasing significance of lysosomal storage diseases, arrangement of awareness programs by governments and the launch of new products are attributed to the growth of the global lysosomal storage diseases therapeutics market. For instance, on May 11, 2023, Chiesi USA, Inc., a U.S. based pharmaceutical company announced that its product Elfabrio was approved by U.S. Food and Drug Administration. It uses the enzyme pegunigalsidase alfa for enzyme replacement therapy (ERT) to treat adults with confirmed Fabry disease.

Key features of the study:

This report provides an in-depth analysis of the global lysosomal storage diseases therapeutics market and provides market size (US$ Billion) and compound annual growth rate (CAGR) for the forecast period (2023-2030), considering 2022 as the base year
It elucidates potential revaenue opportunities across different segments and explains attractive investment proposition matrices for this market
This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
It profiles key players in the global lysosomal storage diseases therapeutics market based on the following parameters-Company highlights, products portfolio, key highlights, financial performance, and strategies
Key companies covered as a part of this study include Shire plc, Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus Therapeutics, Inc.
Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
Global lysosomal storage diseases therapeutics market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global lysosomal storage diseases therapeutics market.

Detailed Segmentation:

Global Lysosomal Storage Diseases Therapeutics Market, By Treatment:
- Enzyme Replacement Therapy
- Stem Cell Therapy
- Substrate Reduction Therapy
- Others
Global Lysosomal Storage Diseases Therapeutics Market, By Indication:
- Gaucher's Disease
- Fabry Disease
- Pompe's Syndrome
- Mucopolysaccharidosis
- Others
Global Lysosomal Storage Diseases Therapeutics Market, By End User:
- Hospitals
- Clinics
Global Lysosomal Storage Diseases Therapeutics Market, By Geography:
- North America
  - By Country:
- U.S.
- Canada
- Europe
- By Country:
- U.K.
- Germany
- Italy
- France
- Spain
- Russia
- Rest of Europe
- Asia Pacific
  - By Country:
- China
- India
- Japan
- ASEAN
- Australia
- South Korea
- Rest of Asia Pacific
- Latin America
  - By Country:
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- Middle East:
  - By Country:
- GCC
- Israel
- Rest of Middle East
- Africa
  - By Country/Region:
- Central Africa
- South Africa
- North Africa
Company Profiles
- Shire plc*
  - Company Overview
  - Product Portfolio
  - Financial Performance
  - Key Strategies
  - Recent Developments
- Pfizer, Inc.
- Sanofi SA
- BioMarin Pharmaceutical Inc
- Actelion Ltd.
- Raptor Pharmaceutical Corp.
- Protalix Biotherapeutics Inc.
- Amicus Therapeutics, Inc.

"*" marked represents similar segmentation in other categories in the respective section.

1. Research Objectives and Assumptions

Research Objectives
Assumptions
Abbreviations

2. Market Purview

Report Description
- Market Definition and Scope
Executive Summary
- Market Snapshot, By Treatment
- Market Snapshot, By Indication
- Market Snapshot, By End user
- Market Snapshot, By Region
Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

Market Dynamics
- Drivers
- Restraints
- Market Opportunities
Impact Analysis
Recent Product Launches
Epidemiology
Merger, Acquisition, and Collaborations
Regulatory Scenario
Key Developments
PEST Analysis

4. Global Lysosomal Storage Diseases Therapeutics Market- COVID-19 Impact Analysis

Economic Impact
COVID-19 Epidemiology
Impact on Supply and Demand

5. Global Lysosomal Storage Diseases Therapeutics Market, By Treatment, 2018-2030, (US$ Billion)

Introduction
- Market Share Analysis, 2023 and 2030 (%)
- Y-o-Y Growth Analysis, 2018 -2030
- Segment Trends
Enzyme Replacement Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Stem Cell Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Substrate Reduction Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)

6. Global Lysosomal Storage Diseases Therapeutics Market, By Indication, 2018-2030, (US$ Billion)

Introduction
- Market Share Analysis, 2023 and 2030 (%)
- Y-o-Y Growth Analysis, 2018 -2030
- Segment Trends
Gaucher's Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Fabry Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Pompe's Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Mucopolysaccharidosis
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)

7. Global Lysosomal Storage Diseases Therapeutics Market, By End user, 2018-2030, (US$ Billion)

Introduction
- Market Share Analysis, 2023 and 2030 (%)
- Y-o-Y Growth Analysis, 2018 -2030
- Segment Trends
Hospitals
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)
Clinics
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2018 - 2030, (US$ Bn)

8. Global Lysosomal Storage Diseases Therapeutics Market, By Region, 2018-2030, (US$ Billion)

Introduction
- Market Share Analysis, By Region, 2023 and 2030 (%)
- Y-o-Y Growth Analysis, For Regions, 2018 -2030
- Regional Trends
North America
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2018 - 2030, (US$ Billion)
U.S.
Canada
Latin America
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2018- 2030, (US$ Billion)
Brazil
Mexico
Argentina
Rest of Latin America
Europe
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2018- 2030, (US$ Billion)
U.K.
Germany
Italy
France
Spain
Russia
Rest of Europe
Asia Pacific
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication , 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2018- 2030, (US$ Billion)
China
India
Japan
ASEAN
Australia
South Korea
Rest of Asia Pacific
Middle East
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2018- 2030, (US$ Billion)
GCC
Israel
Rest of Middle East
Africa
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2018 - 2030, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2018- 2030, (US$ Billion)
North Africa
Central Africa
South Africa

9. Competitive Landscape

Shire plc
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
Pfizer, Inc.
Sanofi SA
BioMarin Pharmaceutical Inc
Actelion Ltd.
Raptor Pharmaceutical Corp
Protalix Biotherapeutics Inc.
Amicus Therapeutics, Inc.

10. Section

Research Methodology
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溶?体贮积病治疗药市场:各治疗药物，各适应症，各终端用户，各地区- 市场规模，占有率，展望，机会分析，2023年～2030年