全球Allan-Herndon-Dudley症候群治療市場：依治療類型、最終用戶、地區

全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）市場規模預計將從2023年的 1,200 萬美元增至2030年的 1,750 萬美元，預測期內的年複合成長率（CAGR）為5.5%。

圖 1.2023年Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺陷）治療的全球市場佔有率（%），依地區分類

Allan-Herndon-Dudley症候群（AHDS）是一種由 MCT8 基因突變引起的罕見遺傳疾病。這些突變導致甲狀腺激素穿過血腦障壁的運輸受損。目前 AHDS 的治療選擇有限，且目的是控制症狀。第一線治療是使用三碘甲狀腺原氨酸（T3）進行甲狀腺荷爾蒙補充療法。 T3補充品可以幫助恢復大腦中的甲狀腺激素濃度並改善神經發育和功能。然而，高劑量的T3有時會引起副作用，如震顫、過動症和心律不整。另一種選擇是甲狀腺素（T4）療法，它依賴 T4 在細胞內外周轉化為活性 T3。這種間接治療方法效果較差，因為 MCT8 突變會抑制荷爾蒙轉運和活化。

新興的基因療法是一種有前景的研究方法，可以直接治療 AHDS 的潛在遺傳原因。基因治療包括將 MCT8 基因的正常副本引進患者的細胞或組織中以替換突變基因。儘管仍處於研究的早期階段，但這可以糾正激素運輸缺陷並消除終身甲狀腺替代方案的需要。挑戰包括開發有效且安全的基因轉移載體，以及根據特定的 MCT8 突變為個別患者量身定做治療方案。進一步的研究進行中，以檢驗基因療法作為未來 AHDS 的創新治療方法。

市場動態：

全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺陷）治療市場主要是由 AHDS 盛行率不斷上升推動的，預計這將在預測期內推動市場成長。例如，根據美國國家生物技術資訊中心（NCBI）2020年1月16日發布的資料，在大約15年的時間裡，已有超過160人被確認受到影響，這意味著這種症候群比之前想像的更為普遍。

研究的主要特點

• 本報告對全球Allan Herndon-Dudley症候群（AHDS）（MCT8缺陷）治療藥物市場進行了詳細分析，並提供了以2022年為基準年的預測期（2023-2030）的市場規模和年複合成長率.（年複合成長率%）。
• 強調了各個細分市場的潛在收益成長機會，並為該市場說明了一系列有吸引力的投資提案。
• 提供了有關市場促進因素、限制因素、機會、新產品發布和核准、市場趨勢、區域前景、主要企業採取的競爭策略等的重要見解。
• 根據公司亮點、產品系列、主要亮點，實績和策略等參數，對全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）治療市場的主要企業進行了分析。
• 本報告的見解使行銷人員和公司負責人能夠就未來的產品發布、升級、市場擴張和行銷策略做出明智的決策。
• 全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）治療市場報告針對該行業的各種相關人員，如投資者、供應商、產品製造商、經銷商、新進入者和財務分析師。
• 相關人員可以透過用於分析全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）治療市場的各種策略矩陣來促進他們的決策。

目錄

第1章 研究目的與前提

• 研究目標
• 先決條件
• 簡稱

第2章 市場展望

• 報告說明
  • 市場定義和範圍
• 執行摘要
• Coherent Opportunity Map（COM）

第3章 市場動態、法規及趨勢分析

• 市場動態
  • AHDS 盛行率增加
  • 全球醫療保健支出增加
• 主要亮點
• 監管場景
• 最近的趨勢
• PEST分析
• 波特的分析
• 合併、收購和合作

第4章 全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）治療市場 - 冠狀病毒（COVID-19）大流行的影響

• 新型冠狀病毒感染疾病（COVID-19）的流行病學
• 供需面分析
• 經濟影響

第5章 全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）治療市場，依治療類型，2018-2030年

• 基因治療
• 荷爾蒙療法

第6章 全球Allan-Herndon-Dudley症候群（AHDS）（MCT8 缺乏症）治療市場，依最終用戶分類，2018-2030年

• 醫院
• 專科診所
• 其他（調查機構等）

第7章 全球Allan-Herndon-Dudley症候群（AHDS）（MCT8缺乏症）治療市場，依地區，2018-2030年

• 北美洲
• 美國
• 加拿大
• 歐洲
• 德國
• 英國
• 西班牙
• 法國
• 義大利
• 俄羅斯
• 其他歐洲國家
• 亞太地區
• 中國
• 印度
• 日本
• 澳洲
• 韓國
• ASEAN
• 其他亞太地區
• 拉丁美洲
• 巴西
• 阿根廷
• 墨西哥
• 其他拉丁美洲
• 中東
• 海灣合作理事會國家
• 以色列
• 其他中東地區
• 非洲
• 北非
• 中部非洲
• 南非

第8章 競爭形勢

• 公司簡介
  • Rare Thyroid Therapeutics International AB
  • Erasmus Medical Center

第9章 章節

• 參考
• 調查方法
• 關於出版商

The global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) market size is expected to reach US$ 17.5 Mn by 2030, from US$ 12 Mn in 2023, exhibiting a compound annual growth rate (CAGR) of 5.5% during the forecast period.

Figure 1. Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market Share (%), By Region, 2023

Allan-Herndon-Dudley syndrome (AHDS) is a rare genetic disorder caused by mutations in the MCT8 gene. These mutations result in impaired thyroid hormone transport across the blood-brain barrier. Current treatment options for AHDS are limited and aim to manage symptoms. The first line of treatment involves thyroid hormone replacement therapy with triiodothyronine (T3). T3 supplements help restore thyroid hormone levels in the brain to improve neurological development and function. However, high T3 doses sometimes cause side effects like tremors, hyperactivity, and irregular heartbeat. The other option is thyroxine (T4) therapy which relies on the peripheral conversion of T4 to active T3 within cells. This indirect method of treatment is not very effective as the MCT8 mutations disrupt hormone transportation and activation.

Emerging gene therapy is a promising investigational approach to directly treat the underlying genetic cause of AHDS. In gene therapy, a normal copy of the MCT8 gene would be delivered into the patient's cells and tissues to replace the mutated one. Though still in early research phase, this could correct the hormone transport defect and eliminate the need for lifelong thyroid supplementation. Challenges include developing effective and safe gene delivery vectors and treatments needing to be tailored to individual patients based on their specific MCT8 mutations. More research is underway to validate gene therapy as an innovative treatment for AHDS in the future.

Market Dynamics:

The global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) treatment market is mainly driven by the increasing prevalence of AHDS is expected to propel the market growth over the forecast period. For instance, according to the data published by the National Center for Biotechnology Information (NCBI) in January 16, 2020, the identification of more than 160 affected individuals in approximately 15 years suggests that the syndrome is more common than previously thought.

Key features of the study:

• This report provides an in-depth analysis of the global allan-herndon-dudley syndrome (AHDS) (MCT8 deficiency) treatment market, and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2023-2030), considering 2022 as the base year
• It elucidates potential revenue growth 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 approvals, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) treatment 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 Rare Thyroid Therapeutics International AB and Erasmus Medical Center
• 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
• The global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) treatment 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 Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) treatment market

Detailed Segmentation:

• Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market, By Treatment Type
  • Gene Therapy
  • Hormone Therapy
• Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market, By End User
  • Hospital
  • Specialty Clinics
  • Others (Research institutes, etc.)
• Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market, By Region
  • North America
  • Europe
  • Asia Pacific
  • Middle East and Africa
  • Latin America
• Top Companies in the Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market:
  • Rare Thyroid Therapeutics International AB
  • Erasmus Medical Center

Table of Contents

1. Research Objectives and Assumptions

• Research Objectives
• Assumptions
• Abbreviations

2. Market Purview

• Report Description
  • Market Definition and Scope
• Executive Summary
  • Market Snippet, By Treatment Type
  • Market Snippet, By End User
  • Market Snippet, By Region
• Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

• Market Dynamics
  • Increasing prevalence of AHDS
  • Increasing Healthcare Expenditure Globally
• Key Highlights
• Regulatory Scenario
• Recent Trends
• PEST Analysis
• PORTER's Analysis
• Mergers, Acquisitions, and Collaborations

4. Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market - Impact of Coronavirus (COVID-19) Pandemic

• COVID-19 Epidemiology
• Supply Side and Demand Side Analysis
• Economic Impact

5. Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market, By Treatment Type, 2018-2030, (US$ Mn)

• Introduction
  • Market Share Analysis, 2023 and 2030 (%)
  • Y-o-Y Growth Analysis, 2019 - 2030
  • Segment Trends
• Gene Therapy
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2018-2030,(US$ Mn)
• Hormone Therapy
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2018-2030,(US$ Mn)

6. Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market, By End User, 2018-2030, (US$ Mn)

• Introduction
  • Market Share Analysis, 2023 and 2030 (%)
  • Y-o-Y Growth Analysis, 2019 - 2030
  • Segment Trends
• Hospital
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2018-2030,(US$ Mn)
• Specialty Clinics
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2018-2030,(US$ Mn)
• Others (Research institutes, etc.)
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2018-2030,(US$ Mn)

7. Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market, By Region, 2018-2030, (US$ Mn)

• Introduction
  • Market Share Analysis, By Region, 2023 and 2030 (%)
  • Y-o-Y Growth Analysis, For Region, 2019 - 2030
  • Country Trends
• North America
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By End User, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2018-2030,(US$ Mn)
• U.S.
• Canada
• Europe
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By End User, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2018-2030,(US$ Mn)
• Germany
• U.K.
• Spain
• France
• Italy
• Russia
• Rest of Europe
• Asia Pacific
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By End User, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2018-2030,(US$ Mn)
• China
• India
• Japan
• Australia
• South Korea
• ASEAN
• Rest of Asia Pacific
• Latin America
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By End User, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2018-2030,(US$ Mn)
• Brazil
• Argentina
• Mexico
• Rest of Latin America
• Middle East
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By End User, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2018-2030,(US$ Mn)
• GCC Countries
• Israel
• Rest of Middle East
• Africa
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By End User, 2018-2030,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Region/Country, 2018-2030,(US$ Mn)
• North Africa
• Central Africa
• South Africa

8. Competitive Landscape

• Company Profile
  • Rare Thyroid Therapeutics International AB
• Company Highlights
• Product Portfolio
• Key Highlights
• Financial Performance
• Strategies
  • Erasmus Medical Center
• Company Highlights
• Product Portfolio
• Key Highlights
• Financial Performance
• Strategies
• Analyst Views

9. Section

• References
• Research Methodology
• About us