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1967659

韓特氏症治療市場－全球產業規模、佔有率、趨勢、機會、預測：按治療方法、最終用戶、地區和競爭格局分類，2021-2031年

Hunter Syndrome Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Treatment, By End User, By Region & Competition, 2021-2031F
出版日期: | 出版商: TechSci Research | 英文 180 Pages | 商品交期: 2-3個工作天內

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簡介目錄

全球韓特氏症治療市場預計將從 2025 年的 14.1 億美元成長到 2031 年的 19.4 億美元，複合年成長率為 5.46%。

此領域專注於開發和推廣治療性介入（包括酵素替代療法和新興基因療法），以治療黏多醣症II型（一種由艾膦酸-2-硫酸酯酶缺乏引起的罕見遺傳性疾病）。推動此領域發展的因素眾多，例如新生兒篩檢計畫的廣泛應用促進了早期療育，以及有利於降低研發財務風險的法規結構（如孤兒藥認定）。這種發展勢頭也體現在罕見疾病領域的活躍度上。美國製藥商協會（PhRMA）在2024年報告稱，監管機構已核准七種用於治療罕見疾病的新型細胞和基因療法，顯示下一代生物製藥的臨床環境良好。

市場概覽
預測期 2027-2031
市場規模：2025年 14.1億美元
市場規模：2031年 19.4億美元
複合年成長率：2026-2031年 5.46%
成長最快的細分市場 酵素替代療法
最大的市場 北美洲

然而，藥物難以穿過血腦障壁這一生理難題，對市場擴張構成了重大障礙。酵素替代療法作為目前的標準療法，往往無法緩解與嚴重神經系統疾病相關的進行性性認知衰退，限制了其臨床療效，也限制了現有產品能夠有效治療的患者群體。

市場促進因素

基因治療和基因組編輯技術的進步正在改變臨床格局，為黏黏多醣症II型（MPSII）的潛在根治性治療方法提供了可能。與慢性管理療法不同，這些治療方法旨在恢復功能性硫酸乙醯肝素酶-2（idronase-2-sulfatase）的產生，從而減輕疾病的系統性負擔，並驗證高價值的評估模型。這一轉變得益於優先考慮罕見疾病的法規環境。美國食品藥物管理局（FDA）報告稱，2024年核准的55種新藥中有28種是用於治療上年度疾病，凸顯了加速藥物研發的努力。例如，Regenxbio公司在2024年9月發布的新聞稿顯示，接受確定劑量RGX-121治療的患者腦脊髓液中硫酸乙醯肝素D2S6的中位以金額為準降低了85%，證實了該療法的療效。

同時，血腦障壁穿越機制的創新解決了傳統靜脈酵素替代療法一直無法克服的難題：無法治療嚴重的神經病變症狀。新興技術利用受體介導的轉運，主動將大分子治療藥物遞送至受影響的中樞神經系統組織，從而實現預防進行性認知衰退的關鍵。正如Denali Therapeutics公司在2024年9月的新聞稿中所述，cibidenofusp alfa的I/II期臨床試驗顯示，到第24週，腦脊髓液中血腦障壁平均降低了90%，這表明全身治療可以有效治療以前被認為無法治癒的神經系統症狀。

市場挑戰

治療藥物生理上無法穿過血腦障壁，是韓特氏症治療全球市場成長的主要障礙。目前，標準療法—酵素替代療法—由於其龐大的分子結構阻礙了其滲透到中樞神經系統，因此對重度神經系統黏多醣症II型（韓特氏症）患者出現的進行性認知功能下降無效。這項限制造成了市場分化：一方面，身體症狀得到控制；另一方面，神經系統疾病持續存在。這嚴重限制了現有生物製藥的商業性潛力，因為最需要介入的患者無法獲得全面的治療。

這項科學挑戰阻礙了市場成長，因為現有治療方案無法滿足巨大的未滿足需求。設計能夠繞過生理屏障的大分子藥物十分複雜，不僅延緩了研發進度，也增加了生產商的風險。根據美國製藥商協會（PhRMA）統計，截至2024年，該產業共有148種用於治療遺傳性疾病的藥物正在研發中，但開發能夠克服中樞神經系統屏障的治療方法仍是研發領域的一大瓶頸。大量在研藥物與缺乏腦滲透性核准之間存在的巨大差距，凸顯了血腦障壁如何持續限制市場發展和商業性擴充性。

市場趨勢

體外幹細胞基因治療方法的發展正在創造一種獨特的治療層級，其療效超越了傳統的體內病毒載體療法。此策略包括改造自體造血幹細胞，使其表達艾膦酸-2-硫酸酯酶，誘導其分化為小膠質細胞，並將該酶直接遞送至大腦，從而實現中樞神經系統的持續修復，同時降低全身免疫抗原性的風險。倫敦大學學院於2025年11月發表的一篇論文詳細闡述了這項發現：接受這種新型Exavio療法的患者在移植後9個月，其語言能力、運動能力和認知發育均顯著改善，展現出阻止神經退化進展的潛力。

此外，神經絲輕鏈生物標記在臨床試驗中的引入，正將檢驗重點從代謝基材轉向神經元健康的直接測量。驗證神經保護作用對於神經病理性表現型的治療方法至關重要，而神經絲輕鏈可提供軸突損傷的量化指標，從而比長期認知功能評估更快地評估療效。根據納斯達克2025年2月的一篇報導，一項tividenofusp alfa的I/II期臨床試驗顯示，在中位追蹤兩年後，神經絲輕鏈水平顯著且持續下降，證實了該生物標記在證明臨床價值方面的實用性。

目錄

第1章概述

第2章：調查方法

第3章執行摘要

第4章：客戶心聲

第5章：韓特氏症治療的全球市場展望

  • 市場規模及預測
    • 按金額
  • 市佔率及預測
    • 依治療方法（酵素替代療法、造血幹細胞移植）
    • 依最終使用者（醫院/診所、門診中心、其他）分類
    • 按地區
    • 按公司（2025 年）
  • 市場地圖

第6章：北美韓特氏症治療市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 北美洲：國別分析
    • 美國
    • 加拿大
    • 墨西哥

第7章：歐洲韓特氏症治療市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 歐洲：國別分析
    • 德國
    • 法國
    • 英國
    • 義大利
    • 西班牙

第8章：亞太地區韓特氏症治療市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 亞太地區：國別分析
    • 中國
    • 印度
    • 日本
    • 韓國
    • 澳洲

第9章：中東和非洲韓特氏症治療市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 中東與非洲：國別分析
    • 沙烏地阿拉伯
    • 阿拉伯聯合大公國
    • 南非

第10章：南美洲韓特氏症治療市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 南美洲：國別分析
    • 巴西
    • 哥倫比亞
    • 阿根廷

第11章 市場動態

  • 促進因素
  • 任務

第12章 市場趨勢與發展

  • 併購
  • 產品發布
  • 近期趨勢

第13章：全球韓特氏症治療市場：SWOT分析

第14章：波特五力分析

  • 產業競爭
  • 新進入者的潛力
  • 供應商的議價能力
  • 顧客權力
  • 替代品的威脅

第15章 競爭格局

  • Takeda Pharmaceutical Co Ltd
  • F Hoffmann-La Roche AG
  • Abbott Laboratories Inc
  • Johnson & Johnson
  • GSK PLC
  • Bayer AG
  • Novartis AG
  • Pfizer Inc
  • Zimmer Biomet Holdings Inc
  • Sangamo Therapeutics, Inc

第16章 策略建議

第17章：關於研究公司及免責聲明

簡介目錄
Product Code: 19231

The Global Hunter Syndrome Treatment Market is projected to expand from USD 1.41 Billion in 2025 to USD 1.94 Billion by 2031, registering a CAGR of 5.46%. This sector focuses on the development and distribution of therapeutic interventions, including enzyme replacement therapies and emerging gene-based treatments, designed to manage Mucopolysaccharidosis II, a rare genetic disorder resulting from iduronate-2-sulfatase deficiency. Growth is underpinned by robust drivers such as the wider adoption of newborn screening programs facilitating early intervention and favorable regulatory frameworks, like orphan drug designations, that reduce R&D financial risks. This momentum is reflected in the rare disease sector's activity; the Pharmaceutical Research and Manufacturers of America noted in 2024 that regulatory agencies approved seven new cell and gene therapies for rare conditions, indicating a clinical environment conducive to next-generation biologics.

Market Overview
Forecast Period2027-2031
Market Size 2025USD 1.41 Billion
Market Size 2031USD 1.94 Billion
CAGR 2026-20315.46%
Fastest Growing SegmentEnzyme Replacement Therapy
Largest MarketNorth America

However, market expansion faces a substantial obstacle regarding the physiological difficulty of delivering therapeutics across the blood-brain barrier. Current standard-of-care enzyme replacement therapies frequently fail to mitigate the progressive cognitive decline linked to the severe, neuronopathic form of the disease, which limits clinical efficacy and restricts the patient population that can be effectively treated with existing products.

Market Driver

Advancements in gene therapy and genome editing are transforming the clinical landscape by providing potential one-time curative treatments that target the genetic origins of Mucopolysaccharidosis II. Unlike chronic management options, these modalities seek to restore functional iduronate-2-sulfatase production, thereby reducing the disease's systemic burden and validating high-value valuation models. This transition is bolstered by a regulatory environment prioritizing orphan conditions; the U.S. Food and Drug Administration reported that 28 of the 55 novel drugs approved in the previous year were for rare diseases in 2024, underscoring a commitment to expediting development. Clinical progress is illustrated by Regenxbio Inc.'s September 2024 press release, which reported that patients receiving the pivotal dose of RGX-121 achieved a median 85% reduction in cerebrospinal fluid heparan sulfate D2S6, confirming the approach's therapeutic viability.

Simultaneously, innovations in blood-brain barrier delivery mechanisms are addressing the historical limitations of intravenous enzyme replacement therapies, which traditionally fail to treat the disorder's severe neuronopathic symptoms. Emerging technologies are utilizing receptor-mediated transport to actively ferry large therapeutic molecules across the blood-brain barrier to reach affected central nervous system tissues, a capability vital for preventing progressive cognitive decline. As reported in a September 2024 press release by Denali Therapeutics Inc., a Phase 1/2 study of tividenofusp alfa demonstrated a 90% mean reduction in cerebrospinal fluid heparan sulfate by Week 24, proving that systemic treatments can effectively target neurological symptoms previously deemed untreatable.

Market Challenge

The physiological inability of therapeutic agents to traverse the blood-brain barrier constitutes a major obstacle to the growth of the Global Hunter Syndrome Treatment Market. Standard-of-care enzyme replacement therapies currently feature large molecular structures that prevent central nervous system penetration, rendering them ineffective against the progressive cognitive decline seen in the severe, neuronopathic form of Mucopolysaccharidosis II. This limitation creates a bifurcated market where somatic symptoms are managed while neurological pathology persists, significantly capping the commercial potential of existing biologics by failing to provide a comprehensive cure for the patients most in need of intervention.

This scientific hurdle restricts market growth by perpetuating a high level of unmet clinical need that current portfolios cannot satisfy. The complexity involved in engineering large-molecule drugs to bypass physiological barriers retards development timelines and elevates the risk profile for manufacturers. According to the Pharmaceutical Research and Manufacturers of America, although the industry had 148 medicines in development for genetic disorders in 2024, successfully formulating therapies that can overcome central nervous system barriers remains a significant operational bottleneck. This gap between broad pipeline activity and the scarcity of brain-penetrant approvals highlights how the blood-brain barrier continues to limit the advancement and commercial scalability of the market.

Market Trends

The development of Ex Vivo Stem Cell Gene Therapy approaches is creating a distinct therapeutic class that operates beyond standard in vivo viral vector modalities. This strategy involves modifying autologous hematopoietic stem cells to express iduronate-2-sulfatase, enabling them to differentiate into microglia and deliver enzymes directly to the brain, which mitigates systemic immunogenicity risks while ensuring durable central nervous system correction. As detailed in a November 2025 article by University College London, a patient treated with this novel ex vivo therapy exhibited dramatic improvements in speech, agility, and cognitive development nine months post-transplant, validating its potential to arrest neurodegenerative progression.

Additionally, the integration of Neurofilament Light Chain biomarkers in clinical trials is shifting analytical focus from metabolic substrates to direct measures of neuronal health. Validating neuroprotection is becoming essential for therapies targeting the neuronopathic phenotype, with neurofilament light chain providing a quantifiable metric of axonal damage that offers a more immediate readout of efficacy than longitudinal cognitive assessments. According to a February 2025 Nasdaq article, a Phase 1/2 study of tividenofusp alfa demonstrated a robust, sustained reduction in neurofilament light chain levels after a median follow-up of two years, confirming the utility of this biomarker in substantiating clinical value.

Key Market Players

  • Takeda Pharmaceutical Co Ltd
  • F Hoffmann-La Roche AG
  • Abbott Laboratories Inc
  • Johnson & Johnson
  • GSK PLC
  • Bayer AG
  • Novartis AG
  • Pfizer Inc
  • Zimmer Biomet Holdings Inc
  • Sangamo Therapeutics, Inc

Report Scope

In this report, the Global Hunter Syndrome Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Hunter Syndrome Treatment Market, By Treatment

  • Enzyme Replacement Therapy
  • Hematopoietic Stem Cell Transplant

Hunter Syndrome Treatment Market, By End User

  • Hospitals & Clinics
  • Ambulatory Care Centers
  • Others

Hunter Syndrome Treatment Market, By Region

  • North America
    • United States
    • Canada
    • Mexico
  • Europe
    • France
    • United Kingdom
    • Italy
    • Germany
    • Spain
  • Asia Pacific
    • China
    • India
    • Japan
    • Australia
    • South Korea
  • South America
    • Brazil
    • Argentina
    • Colombia
  • Middle East & Africa
    • South Africa
    • Saudi Arabia
    • UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Hunter Syndrome Treatment Market.

Available Customizations:

Global Hunter Syndrome Treatment Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

  • Detailed analysis and profiling of additional market players (up to five).

Table of Contents

1. Product Overview

  • 1.1. Market Definition
  • 1.2. Scope of the Market
    • 1.2.1. Markets Covered
    • 1.2.2. Years Considered for Study
    • 1.2.3. Key Market Segmentations

2. Research Methodology

  • 2.1. Objective of the Study
  • 2.2. Baseline Methodology
  • 2.3. Key Industry Partners
  • 2.4. Major Association and Secondary Sources
  • 2.5. Forecasting Methodology
  • 2.6. Data Triangulation & Validation
  • 2.7. Assumptions and Limitations

3. Executive Summary

  • 3.1. Overview of the Market
  • 3.2. Overview of Key Market Segmentations
  • 3.3. Overview of Key Market Players
  • 3.4. Overview of Key Regions/Countries
  • 3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Global Hunter Syndrome Treatment Market Outlook

  • 5.1. Market Size & Forecast
    • 5.1.1. By Value
  • 5.2. Market Share & Forecast
    • 5.2.1. By Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Transplant)
    • 5.2.2. By End User (Hospitals & Clinics, Ambulatory Care Centers, Others)
    • 5.2.3. By Region
    • 5.2.4. By Company (2025)
  • 5.3. Market Map

6. North America Hunter Syndrome Treatment Market Outlook

  • 6.1. Market Size & Forecast
    • 6.1.1. By Value
  • 6.2. Market Share & Forecast
    • 6.2.1. By Treatment
    • 6.2.2. By End User
    • 6.2.3. By Country
  • 6.3. North America: Country Analysis
    • 6.3.1. United States Hunter Syndrome Treatment Market Outlook
      • 6.3.1.1. Market Size & Forecast
        • 6.3.1.1.1. By Value
      • 6.3.1.2. Market Share & Forecast
        • 6.3.1.2.1. By Treatment
        • 6.3.1.2.2. By End User
    • 6.3.2. Canada Hunter Syndrome Treatment Market Outlook
      • 6.3.2.1. Market Size & Forecast
        • 6.3.2.1.1. By Value
      • 6.3.2.2. Market Share & Forecast
        • 6.3.2.2.1. By Treatment
        • 6.3.2.2.2. By End User
    • 6.3.3. Mexico Hunter Syndrome Treatment Market Outlook
      • 6.3.3.1. Market Size & Forecast
        • 6.3.3.1.1. By Value
      • 6.3.3.2. Market Share & Forecast
        • 6.3.3.2.1. By Treatment
        • 6.3.3.2.2. By End User

7. Europe Hunter Syndrome Treatment Market Outlook

  • 7.1. Market Size & Forecast
    • 7.1.1. By Value
  • 7.2. Market Share & Forecast
    • 7.2.1. By Treatment
    • 7.2.2. By End User
    • 7.2.3. By Country
  • 7.3. Europe: Country Analysis
    • 7.3.1. Germany Hunter Syndrome Treatment Market Outlook
      • 7.3.1.1. Market Size & Forecast
        • 7.3.1.1.1. By Value
      • 7.3.1.2. Market Share & Forecast
        • 7.3.1.2.1. By Treatment
        • 7.3.1.2.2. By End User
    • 7.3.2. France Hunter Syndrome Treatment Market Outlook
      • 7.3.2.1. Market Size & Forecast
        • 7.3.2.1.1. By Value
      • 7.3.2.2. Market Share & Forecast
        • 7.3.2.2.1. By Treatment
        • 7.3.2.2.2. By End User
    • 7.3.3. United Kingdom Hunter Syndrome Treatment Market Outlook
      • 7.3.3.1. Market Size & Forecast
        • 7.3.3.1.1. By Value
      • 7.3.3.2. Market Share & Forecast
        • 7.3.3.2.1. By Treatment
        • 7.3.3.2.2. By End User
    • 7.3.4. Italy Hunter Syndrome Treatment Market Outlook
      • 7.3.4.1. Market Size & Forecast
        • 7.3.4.1.1. By Value
      • 7.3.4.2. Market Share & Forecast
        • 7.3.4.2.1. By Treatment
        • 7.3.4.2.2. By End User
    • 7.3.5. Spain Hunter Syndrome Treatment Market Outlook
      • 7.3.5.1. Market Size & Forecast
        • 7.3.5.1.1. By Value
      • 7.3.5.2. Market Share & Forecast
        • 7.3.5.2.1. By Treatment
        • 7.3.5.2.2. By End User

8. Asia Pacific Hunter Syndrome Treatment Market Outlook

  • 8.1. Market Size & Forecast
    • 8.1.1. By Value
  • 8.2. Market Share & Forecast
    • 8.2.1. By Treatment
    • 8.2.2. By End User
    • 8.2.3. By Country
  • 8.3. Asia Pacific: Country Analysis
    • 8.3.1. China Hunter Syndrome Treatment Market Outlook
      • 8.3.1.1. Market Size & Forecast
        • 8.3.1.1.1. By Value
      • 8.3.1.2. Market Share & Forecast
        • 8.3.1.2.1. By Treatment
        • 8.3.1.2.2. By End User
    • 8.3.2. India Hunter Syndrome Treatment Market Outlook
      • 8.3.2.1. Market Size & Forecast
        • 8.3.2.1.1. By Value
      • 8.3.2.2. Market Share & Forecast
        • 8.3.2.2.1. By Treatment
        • 8.3.2.2.2. By End User
    • 8.3.3. Japan Hunter Syndrome Treatment Market Outlook
      • 8.3.3.1. Market Size & Forecast
        • 8.3.3.1.1. By Value
      • 8.3.3.2. Market Share & Forecast
        • 8.3.3.2.1. By Treatment
        • 8.3.3.2.2. By End User
    • 8.3.4. South Korea Hunter Syndrome Treatment Market Outlook
      • 8.3.4.1. Market Size & Forecast
        • 8.3.4.1.1. By Value
      • 8.3.4.2. Market Share & Forecast
        • 8.3.4.2.1. By Treatment
        • 8.3.4.2.2. By End User
    • 8.3.5. Australia Hunter Syndrome Treatment Market Outlook
      • 8.3.5.1. Market Size & Forecast
        • 8.3.5.1.1. By Value
      • 8.3.5.2. Market Share & Forecast
        • 8.3.5.2.1. By Treatment
        • 8.3.5.2.2. By End User

9. Middle East & Africa Hunter Syndrome Treatment Market Outlook

  • 9.1. Market Size & Forecast
    • 9.1.1. By Value
  • 9.2. Market Share & Forecast
    • 9.2.1. By Treatment
    • 9.2.2. By End User
    • 9.2.3. By Country
  • 9.3. Middle East & Africa: Country Analysis
    • 9.3.1. Saudi Arabia Hunter Syndrome Treatment Market Outlook
      • 9.3.1.1. Market Size & Forecast
        • 9.3.1.1.1. By Value
      • 9.3.1.2. Market Share & Forecast
        • 9.3.1.2.1. By Treatment
        • 9.3.1.2.2. By End User
    • 9.3.2. UAE Hunter Syndrome Treatment Market Outlook
      • 9.3.2.1. Market Size & Forecast
        • 9.3.2.1.1. By Value
      • 9.3.2.2. Market Share & Forecast
        • 9.3.2.2.1. By Treatment
        • 9.3.2.2.2. By End User
    • 9.3.3. South Africa Hunter Syndrome Treatment Market Outlook
      • 9.3.3.1. Market Size & Forecast
        • 9.3.3.1.1. By Value
      • 9.3.3.2. Market Share & Forecast
        • 9.3.3.2.1. By Treatment
        • 9.3.3.2.2. By End User

10. South America Hunter Syndrome Treatment Market Outlook

  • 10.1. Market Size & Forecast
    • 10.1.1. By Value
  • 10.2. Market Share & Forecast
    • 10.2.1. By Treatment
    • 10.2.2. By End User
    • 10.2.3. By Country
  • 10.3. South America: Country Analysis
    • 10.3.1. Brazil Hunter Syndrome Treatment Market Outlook
      • 10.3.1.1. Market Size & Forecast
        • 10.3.1.1.1. By Value
      • 10.3.1.2. Market Share & Forecast
        • 10.3.1.2.1. By Treatment
        • 10.3.1.2.2. By End User
    • 10.3.2. Colombia Hunter Syndrome Treatment Market Outlook
      • 10.3.2.1. Market Size & Forecast
        • 10.3.2.1.1. By Value
      • 10.3.2.2. Market Share & Forecast
        • 10.3.2.2.1. By Treatment
        • 10.3.2.2.2. By End User
    • 10.3.3. Argentina Hunter Syndrome Treatment Market Outlook
      • 10.3.3.1. Market Size & Forecast
        • 10.3.3.1.1. By Value
      • 10.3.3.2. Market Share & Forecast
        • 10.3.3.2.1. By Treatment
        • 10.3.3.2.2. By End User

11. Market Dynamics

  • 11.1. Drivers
  • 11.2. Challenges

12. Market Trends & Developments

  • 12.1. Merger & Acquisition (If Any)
  • 12.2. Product Launches (If Any)
  • 12.3. Recent Developments

13. Global Hunter Syndrome Treatment Market: SWOT Analysis

14. Porter's Five Forces Analysis

  • 14.1. Competition in the Industry
  • 14.2. Potential of New Entrants
  • 14.3. Power of Suppliers
  • 14.4. Power of Customers
  • 14.5. Threat of Substitute Products

15. Competitive Landscape

  • 15.1. Takeda Pharmaceutical Co Ltd
    • 15.1.1. Business Overview
    • 15.1.2. Products & Services
    • 15.1.3. Recent Developments
    • 15.1.4. Key Personnel
    • 15.1.5. SWOT Analysis
  • 15.2. F Hoffmann-La Roche AG
  • 15.3. Abbott Laboratories Inc
  • 15.4. Johnson & Johnson
  • 15.5. GSK PLC
  • 15.6. Bayer AG
  • 15.7. Novartis AG
  • 15.8. Pfizer Inc
  • 15.9. Zimmer Biomet Holdings Inc
  • 15.10. Sangamo Therapeutics, Inc

16. Strategic Recommendations

17. About Us & Disclaimer