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市场调查报告书
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1587615

到 2030 年韩特氏症治疗市场预测：按治疗类型、併发症类型、给药途径、严重程度、最终用户和地区进行的全球分析

Hunter Syndrome Treatment Market Forecasts to 2030 - Global Analysis By Treatment Type, Complication Type, Route of Administration, Severity, End User and By Geography
出版日期: | 出版商: Stratistics Market Research Consulting | 英文 200+ Pages | 商品交期: 2-3个工作天内

价格

根据 Stratistics MRC 的数据，2024 年全球韩特氏症治疗市场规模为 13 亿美元，预计在预测期内将以 7.3% 的复合年增长率增长，并在 2030 年达到 19 亿美元。

韩特氏症是一种罕见的遗传性疾病，由艾杜醣醛酸-2-硫酸酯酶缺乏引起，会导致进行性健康问题。治疗主要着重于控制症状和改善生活品质。酵素替代疗法（ERT）是最常见的方法，其中向患者提供缺乏的酶以减少基材积累。其他治疗包括维持运动功能的物理治疗、解决呼吸困难的呼吸护理以及解决整形外科问题和支持器官功能的手术干预。

根据遗传和罕见疾病资讯中心（GARD）统计，估计有 7,000 种罕见疾病，在美国受这些罕见疾病影响的总人数为 25 至 3000 万人。

对韩特氏症的认识不断提高

对韩特氏症的认识不断提高，透过促进早期诊断和更深入地了解疾病，正在重塑治疗市场。加强医疗保健专业人员和倡导团体的教育正在推动对酵素替代疗法 (ERT) 等创新治疗方法的需求。这种认可将促进相关人员之间的合作，改善患者的支持和结果，并最终扩大治疗选择并改善受疾病影响的人们的整体状况。

缺乏熟练的专业人员

市场上缺乏熟练的专业人员是一个重大挑战，导致诊断延迟和患者治疗效果不佳。缺乏专业知识可能会阻碍酵素替代疗法和基因疗法等创新治疗方法的有效实施。这种短缺限制了获得专门治疗的机会，并降低了患者支持和整体护理品质。结果，健康差距加剧，改善该疾病患者预后的潜力也降低。

医疗保健支出增加

市场医疗保健支出的增加反映了对先进治疗和全面护理的需求不断增长。随着认识的提高，对酵素替代疗法（ERT）和基因疗法的投资将会扩大，与研究、开发和患者管理相关的成本将会增加。支出的激增可能会给旨在改善治疗机会和结果的医疗保健预算带来压力。最终，它强调需要永续资金筹措和创新方法来有效支持受影响的人。

新兴市场认知度低

新兴市场对韩特氏症的认识不足阻碍了治疗，导致诊断延迟和获得治疗的机会不足。许多医疗保健提供者缺乏对这种疾病的了解，这可能导致误诊或诊断不足。这一差距限制了创新治疗方法的获得，并最终影响患者的治疗结果。加强教育和宣传对于提高对这些地区受影响人群的认识和支持至关重要。

COVID-19 的影响：

COVID-19 大流行扰乱了医疗保健服务并限制了获得基本治疗方法，从而对市场产生了负面影响。常规就诊被推迟，导致许多患者的诊断和治疗开始延迟。此外，供应链中断也影响了酵素替代疗法的可用性。对大流行应对的关注已经将资源从罕见疾病上转移出来，这凸显了建立有弹性的医疗保健系统的必要性，该系统优先考虑为患有韩特氏症等罕见疾病的患者提供连续性护理。

预计在预测期内，肌肉骨骼部分将是最大的。

预计肌肉骨骼领域在预测期内将占据最大的市场占有率。患者可能需要物理治疗、整形外科手术和疼痛管理策略等干预措施来解决这些併发症。随着我们对肌肉骨骼疾病的了解不断加深，对专业治疗和多学科护理方法的需求不断增长。有效的管理对于改善患者的生活品质和整体功能结果至关重要。

预计诊断中心产业在预测期内复合年增长率最高

预计诊断中心产业在预测期内将呈现最高的复合年增长率。先进的测试方法，包括基因测试和酶测定，对于确认这种疾病至关重要。加强诊断中心和医疗保健提供者之间的合作将提高意识并促进及时介入。随着越来越多的中心专注于罕见遗传疾病，它们将有助于改善患者的治疗效果，增加获得标靶治疗的机会，并最终改善受影响者的护理。

比最大的地区

预计北美地区在预测期内将占据最大的市场占有率。酵素替代疗法 (ERT) 透过持续的临床研究和患者宣传工作正在获得支持。获得专业治疗和诊断服务的机会正在改善，但在费用和保险范围方面仍然存在挑战。医疗保健提供者、製药公司和倡导团体之间的合作对于改善该地区患者的治疗效果至关重要。

复合年增长率最高的地区：

由于遗传病患病率不断上升，预计亚太地区在预测期内将出现最高的成长率。该市场的特点是治疗方式多种多样，包括 ERT、干细胞移植和手术干预，但 ERT 由于其在控制关节活动度和麵部粗糙等症状方面的有效性而目前占据主导地位。日本和中国等国家在这一领域处于领先地位，受益于早期诊断和对罕见疾病认识的提高。

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目录

第一章执行摘要

第二章 前言

  • 概述
  • 相关利益者
  • 调查范围
  • 调查方法
    • 资料探勘
    • 资料分析
    • 资料检验
    • 研究途径
  • 研究资讯来源
    • 主要研究资讯来源
    • 二次研究资讯来源
    • 先决条件

第三章市场趋势分析

  • 促进因素
  • 抑制因素
  • 机会
  • 威胁
  • 最终用户分析
  • 新兴市场
  • COVID-19 的影响

第4章波特五力分析

  • 供应商的议价能力
  • 买方议价能力
  • 替代品的威胁
  • 新进入者的威胁
  • 竞争公司之间的敌对关係

第五章全球韩特氏症治疗市场：依治疗类型

  • 酵素替代疗法（ERT）
  • 造血干细胞移植（HSCT）
  • 基因治疗
  • 其他类型的治疗

第六章韩特氏症治疗的全球市场：依併发症类型

  • 心血管
  • 呼吸系统
  • 肌肉骨骼
  • 神经学的
  • 眼科
  • 肝
  • 肾
  • 血液学
  • 其他併发症类型

第七章全球韩特氏症治疗市场：依给药途径

  • 静脉注射 (IV)
  • 脑室内 (ICV)

第八章全球韩特氏症治疗市场：依严重程度

  • 温和的
  • 缓和
  • 严重

第九章全球韩特氏症治疗市场：依最终用户分类

  • 医院
  • 诊断中心
  • 专科诊所
  • 居家照护环境
  • 其他最终用户

第十章全球韩特氏症治疗市场：按地区

  • 北美洲
    • 美国
    • 加拿大
    • 墨西哥
  • 欧洲
    • 德国
    • 英国
    • 义大利
    • 法国
    • 西班牙
    • 其他欧洲国家
  • 亚太地区
    • 日本
    • 中国
    • 印度
    • 澳洲
    • 纽西兰
    • 韩国
    • 其他亚太地区
  • 南美洲
    • 阿根廷
    • 巴西
    • 智利
    • 南美洲其他地区
  • 中东/非洲
    • 沙乌地阿拉伯
    • 阿拉伯聯合大公国
    • 卡达
    • 南非
    • 其他中东和非洲

第十一章 主要进展

  • 合约、伙伴关係、协作和合资企业
  • 收购和合併
  • 新产品发布
  • 业务拓展
  • 其他关键策略

第十二章 公司概况

  • Takeda Pharmaceutical Company Limited
  • Medtronic
  • CANbridge Life Sciences Ltd.
  • Novartis AG
  • Zimmer Biomet
  • Sangamo Therapeutics
  • F. Hoffmann-La Roche Ltd
  • Jasper Therapeutics, Inc.
  • GSK Plc.
  • Denali Therapeutics
  • Abbott
  • JCR Pharmaceuticals Co., Ltd.
  • Bayer AG
  • Johnson & Johnson Services, Inc.
  • Stryker Corporation
Product Code: SMRC27600

According to Stratistics MRC, the Global Hunter Syndrome Treatment Market is accounted for $1.3 billion in 2024 and is expected to reach $1.9 billion by 2030 growing at a CAGR of 7.3% during the forecast period. Hunter Syndrome, a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase, leads to progressive health issues. Treatment primarily focuses on managing symptoms and improving quality of life. Enzyme replacement therapy (ERT) is the most common approach, providing patients with the missing enzyme to reduce substrate accumulation. Additional treatments may include physical therapy to maintain mobility, respiratory care to address breathing difficulties, and surgical interventions for orthopedic issues or organ function support.

According to the Genetic and Rare Diseases Information Center (GARD), there can be an estimated 7,000 rare diseases and the total number of individuals in the U.S. from these rare diseases can be 25-30 million.

Market Dynamics:

Driver:

Growing awareness of hunter syndrome

Growing awareness of Hunter Syndrome is reshaping the treatment market by promoting earlier diagnoses and a deeper understanding of the condition. Increased education among healthcare professionals and advocacy groups has spurred demand for innovative therapies, such as enzyme replacement therapy (ERT). This awareness fosters collaboration among stakeholders, enhancing patient support and outcomes, ultimately expanding treatment options and improving the overall landscape for those affected by the disorder.

Restraint:

Lack of skilled professionals

The lack of skilled professionals in the market poses significant challenges, leading to delayed diagnoses and suboptimal care for patients. Insufficient expertise can hinder effective implementation of innovative therapies, such as enzyme replacement and gene therapies. This shortage limits access to specialized treatment, reducing patient support and overall quality of care. Consequently, it exacerbates health disparities and diminishes the potential for improved outcomes in individuals affected by the disorder.

Opportunity:

Increased healthcare expenditure

Increased healthcare expenditure in the market reflects the rising demand for advanced therapies and comprehensive care. As awareness grows, investments in enzyme replacement therapy (ERT) and gene therapies expand, driving up costs associated with research, development, and patient management. This surge in spending aims to improve treatment accessibility and outcomes but may strain healthcare budgets. Ultimately, it highlights the need for sustainable funding and innovative approaches to support affected individuals effectively.

Threat:

Limited awareness in emerging markets

Limited awareness of Hunter Syndrome in emerging markets significantly hinders the treatment landscape, resulting in delayed diagnoses and inadequate access to care. Many healthcare providers may lack knowledge about the disorder, leading to misdiagnosis or underdiagnosis. This gap restricts the availability of innovative therapies ultimately impacting patient outcomes. Increased education and advocacy are essential to improve awareness and support for affected individuals in these regions.

Covid-19 Impact:

The COVID-19 pandemic adversely impacted the market by disrupting healthcare services and limiting access to essential therapies. Routine medical visits were postponed, delaying diagnoses and treatment initiation for many patients. Additionally, supply chain disruptions affected the availability of enzyme replacement therapies. The focus on pandemic response shifted resources away from rare diseases, highlighting the need for resilient healthcare systems that prioritize continuity of care for patients with rare conditions like Hunter Syndrome.

The musculoskeletal segment is projected to be the largest during the forecast period

The musculoskeletal segment is projected to account for the largest market share during the projection period. Patients may require interventions such as physical therapy, orthopedic surgeries, and pain management strategies to address these complications. The growing understanding of musculoskeletal manifestations drives demand for specialized treatments and multidisciplinary care approaches. Effective management is crucial to improving patients' quality of life and overall functional outcomes.

The diagnostic centers segment is expected to have the highest CAGR during the forecast period

The diagnostic centers segment is expected to have the highest CAGR during the extrapolated period. Advanced testing methods, including genetic testing and enzyme assays, are essential for confirming the disorder. Increased collaboration between diagnostic centers and healthcare providers enhances awareness and promotes timely intervention. As more centers specialize in rare genetic disorders, they contribute to improved patient outcomes and better access to targeted therapies, ultimately advancing care for those affected.

Region with largest share:

North America region is projected to account for the largest market share during the forecast period. Enzyme replacement therapy (ERT) has gained traction, supported by ongoing clinical research and patient advocacy efforts. Access to specialized care and diagnostic services is improving, although challenges remain in terms of cost and insurance coverage. The collaboration among healthcare providers, pharmaceutical companies, and advocacy groups is crucial for enhancing patient outcomes in the region.

Region with highest CAGR:

Asia Pacific is expected to register the highest growth rate over the forecast period due to increasing prevalence of genetic disorders. The market is characterized by diverse treatment modalities, including ERT, stem cell transplants, and surgical interventions, with ERT currently dominating due to its efficacy in managing symptoms like joint mobility and facial coarseness. Countries such as Japan and China are leading the charge, benefiting from early diagnosis and increased awareness of rare diseases.

Key players in the market

Some of the key players in Hunter Syndrome Treatment market include Takeda Pharmaceutical Company Limited, Medtronic, CANbridge Life Sciences Ltd., Novartis AG, Zimmer Biomet, Sangamo Therapeutics, F. Hoffmann-La Roche Ltd, Jasper Therapeutics, Inc., GSK Plc., Denali Therapeutics, Abbott, JCR Pharmaceuticals Co., Ltd. , Bayer AG, Johnson & Johnson Services, Inc. and Stryker Corporation.

Key Developments:

In September 2024, Denali Therapeutics announced plans to seek accelerated FDA approval for its investigational treatment, DNL310, to address Hunter syndrome (MPS II). Following productive discussions with the FDA, the company is set to submit a Biologics License Application (BLA) by early 2025.

In January 2024, Clinigen Group plc announced that it had received manufacturing and marketing approval for Hunterase (Disulfate-beta) ICV 15mg, under its strategic alliance with GC Pharma in Japan. This approval may help the company to acquire an enhanced customer base.

Treatment Types Covered:

  • Enzyme Replacement Therapy (ERT)
  • Hematopoietic Stem Cell Transplant (HSCT)
  • Gene Therapy
  • Other Treatment Types

Complication Types Covered:

  • Cardiovascular
  • Respiratory
  • Musculoskeletal
  • Neurological
  • Ocular
  • Hepatic
  • Renal
  • Hematological
  • Other Complication Types

Route of Administration Covered:

  • Intravenous (IV)
  • Intracerebroventricular (ICV)

Severity Covered:

  • Mild
  • Moderate
  • Severe

End Users Covered:

  • Hospitals
  • Diagnostic Centers
  • Specialty Clinics
  • Homecare Settings
  • Other End Users

Regions Covered:

  • North America
    • US
    • Canada
    • Mexico
  • Europe
    • Germany
    • UK
    • Italy
    • France
    • Spain
    • Rest of Europe
  • Asia Pacific
    • Japan
    • China
    • India
    • Australia
    • New Zealand
    • South Korea
    • Rest of Asia Pacific
  • South America
    • Argentina
    • Brazil
    • Chile
    • Rest of South America
  • Middle East & Africa
    • Saudi Arabia
    • UAE
    • Qatar
    • South Africa
    • Rest of Middle East & Africa

What our report offers:

  • Market share assessments for the regional and country-level segments
  • Strategic recommendations for the new entrants
  • Covers Market data for the years 2022, 2023, 2024, 2026, and 2030
  • Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations)
  • Strategic recommendations in key business segments based on the market estimations
  • Competitive landscaping mapping the key common trends
  • Company profiling with detailed strategies, financials, and recent developments
  • Supply chain trends mapping the latest technological advancements

Free Customization Offerings:

All the customers of this report will be entitled to receive one of the following free customization options:

  • Company Profiling
    • Comprehensive profiling of additional market players (up to 3)
    • SWOT Analysis of key players (up to 3)
  • Regional Segmentation
    • Market estimations, Forecasts and CAGR of any prominent country as per the client's interest (Note: Depends on feasibility check)
  • Competitive Benchmarking
    • Benchmarking of key players based on product portfolio, geographical presence, and strategic alliances

Table of Contents

1 Executive Summary

2 Preface

  • 2.1 Abstract
  • 2.2 Stake Holders
  • 2.3 Research Scope
  • 2.4 Research Methodology
    • 2.4.1 Data Mining
    • 2.4.2 Data Analysis
    • 2.4.3 Data Validation
    • 2.4.4 Research Approach
  • 2.5 Research Sources
    • 2.5.1 Primary Research Sources
    • 2.5.2 Secondary Research Sources
    • 2.5.3 Assumptions

3 Market Trend Analysis

  • 3.1 Introduction
  • 3.2 Drivers
  • 3.3 Restraints
  • 3.4 Opportunities
  • 3.5 Threats
  • 3.6 End User Analysis
  • 3.7 Emerging Markets
  • 3.8 Impact of Covid-19

4 Porters Five Force Analysis

  • 4.1 Bargaining power of suppliers
  • 4.2 Bargaining power of buyers
  • 4.3 Threat of substitutes
  • 4.4 Threat of new entrants
  • 4.5 Competitive rivalry

5 Global Hunter Syndrome Treatment Market, By Treatment Type

  • 5.1 Introduction
  • 5.2 Enzyme Replacement Therapy (ERT)
  • 5.3 Hematopoietic Stem Cell Transplant (HSCT)
  • 5.4 Gene Therapy
  • 5.5 Other Treatment Types

6 Global Hunter Syndrome Treatment Market, By Complication Type

  • 6.1 Introduction
  • 6.2 Cardiovascular
  • 6.3 Respiratory
  • 6.4 Musculoskeletal
  • 6.5 Neurological
  • 6.6 Ocular
  • 6.7 Hepatic
  • 6.8 Renal
  • 6.9 Hematological
  • 6.10 Other Complication Types

7 Global Hunter Syndrome Treatment Market, By Route of Administration

  • 7.1 Introduction
  • 7.2 Intravenous (IV)
  • 7.3 Intracerebroventricular (ICV)

8 Global Hunter Syndrome Treatment Market, By Severity

  • 8.1 Introduction
  • 8.2 Mild
  • 8.3 Moderate
  • 8.4 Severe

9 Global Hunter Syndrome Treatment Market, By End User

  • 9.1 Introduction
  • 9.2 Hospitals
  • 9.3 Diagnostic Centers
  • 9.4 Specialty Clinics
  • 9.5 Homecare Settings
  • 9.6 Other End Users

10 Global Hunter Syndrome Treatment Market, By Geography

  • 10.1 Introduction
  • 10.2 North America
    • 10.2.1 US
    • 10.2.2 Canada
    • 10.2.3 Mexico
  • 10.3 Europe
    • 10.3.1 Germany
    • 10.3.2 UK
    • 10.3.3 Italy
    • 10.3.4 France
    • 10.3.5 Spain
    • 10.3.6 Rest of Europe
  • 10.4 Asia Pacific
    • 10.4.1 Japan
    • 10.4.2 China
    • 10.4.3 India
    • 10.4.4 Australia
    • 10.4.5 New Zealand
    • 10.4.6 South Korea
    • 10.4.7 Rest of Asia Pacific
  • 10.5 South America
    • 10.5.1 Argentina
    • 10.5.2 Brazil
    • 10.5.3 Chile
    • 10.5.4 Rest of South America
  • 10.6 Middle East & Africa
    • 10.6.1 Saudi Arabia
    • 10.6.2 UAE
    • 10.6.3 Qatar
    • 10.6.4 South Africa
    • 10.6.5 Rest of Middle East & Africa

11 Key Developments

  • 11.1 Agreements, Partnerships, Collaborations and Joint Ventures
  • 11.2 Acquisitions & Mergers
  • 11.3 New Product Launch
  • 11.4 Expansions
  • 11.5 Other Key Strategies

12 Company Profiling

  • 12.1 Takeda Pharmaceutical Company Limited
  • 12.2 Medtronic
  • 12.3 CANbridge Life Sciences Ltd.
  • 12.4 Novartis AG
  • 12.5 Zimmer Biomet
  • 12.6 Sangamo Therapeutics
  • 12.7 F. Hoffmann-La Roche Ltd
  • 12.8 Jasper Therapeutics, Inc.
  • 12.9 GSK Plc.
  • 12.10 Denali Therapeutics
  • 12.11 Abbott
  • 12.12 JCR Pharmaceuticals Co., Ltd.
  • 12.13 Bayer AG
  • 12.14 Johnson & Johnson Services, Inc.
  • 12.15 Stryker Corporation

List of Tables

  • Table 1 Global Hunter Syndrome Treatment Market Outlook, By Region (2022-2030) ($MN)
  • Table 2 Global Hunter Syndrome Treatment Market Outlook, By Treatment Type (2022-2030) ($MN)
  • Table 3 Global Hunter Syndrome Treatment Market Outlook, By Enzyme Replacement Therapy (ERT) (2022-2030) ($MN)
  • Table 4 Global Hunter Syndrome Treatment Market Outlook, By Hematopoietic Stem Cell Transplant (HSCT) (2022-2030) ($MN)
  • Table 5 Global Hunter Syndrome Treatment Market Outlook, By Gene Therapy (2022-2030) ($MN)
  • Table 6 Global Hunter Syndrome Treatment Market Outlook, By Other Treatment Types (2022-2030) ($MN)
  • Table 7 Global Hunter Syndrome Treatment Market Outlook, By Complication Type (2022-2030) ($MN)
  • Table 8 Global Hunter Syndrome Treatment Market Outlook, By Cardiovascular (2022-2030) ($MN)
  • Table 9 Global Hunter Syndrome Treatment Market Outlook, By Respiratory (2022-2030) ($MN)
  • Table 10 Global Hunter Syndrome Treatment Market Outlook, By Musculoskeletal (2022-2030) ($MN)
  • Table 11 Global Hunter Syndrome Treatment Market Outlook, By Neurological (2022-2030) ($MN)
  • Table 12 Global Hunter Syndrome Treatment Market Outlook, By Ocular (2022-2030) ($MN)
  • Table 13 Global Hunter Syndrome Treatment Market Outlook, By Hepatic (2022-2030) ($MN)
  • Table 14 Global Hunter Syndrome Treatment Market Outlook, By Renal (2022-2030) ($MN)
  • Table 15 Global Hunter Syndrome Treatment Market Outlook, By Hematological (2022-2030) ($MN)
  • Table 16 Global Hunter Syndrome Treatment Market Outlook, By Other Complication Types (2022-2030) ($MN)
  • Table 17 Global Hunter Syndrome Treatment Market Outlook, By Route of Administration (2022-2030) ($MN)
  • Table 18 Global Hunter Syndrome Treatment Market Outlook, By Intravenous (IV) (2022-2030) ($MN)
  • Table 19 Global Hunter Syndrome Treatment Market Outlook, By Intracerebroventricular (ICV) (2022-2030) ($MN)
  • Table 20 Global Hunter Syndrome Treatment Market Outlook, By Severity (2022-2030) ($MN)
  • Table 21 Global Hunter Syndrome Treatment Market Outlook, By Mild (2022-2030) ($MN)
  • Table 22 Global Hunter Syndrome Treatment Market Outlook, By Moderate (2022-2030) ($MN)
  • Table 23 Global Hunter Syndrome Treatment Market Outlook, By Severe (2022-2030) ($MN)
  • Table 24 Global Hunter Syndrome Treatment Market Outlook, By End User (2022-2030) ($MN)
  • Table 25 Global Hunter Syndrome Treatment Market Outlook, By Hospitals (2022-2030) ($MN)
  • Table 26 Global Hunter Syndrome Treatment Market Outlook, By Diagnostic Centers (2022-2030) ($MN)
  • Table 27 Global Hunter Syndrome Treatment Market Outlook, By Specialty Clinics (2022-2030) ($MN)
  • Table 28 Global Hunter Syndrome Treatment Market Outlook, By Homecare Settings (2022-2030) ($MN)
  • Table 29 Global Hunter Syndrome Treatment Market Outlook, By Other End Users (2022-2030) ($MN)

Note: Tables for North America, Europe, APAC, South America, and Middle East & Africa Regions are also represented in the same manner as above.