全球莫基奥症候群（MPS-IV）药物市场按产品类型、治疗方法、疾病类型、分销管道和地区划分

2025 年全球 Morquio 症候群 (MPS-IV) 药物市场规模估计为 190 万美元，预计到 2032 年将达到 440 万美元，2025 年至 2032 年的年复合成长率（CAGR）为 12.7%。

数字。 2025 年全球市场占有率（按地区）

黏多醣症IV 型 (MPS IV) 是一种罕见疾病，患者体内缺乏或没有足够的酵素来分解长链糖分子。这些分子链被称为Glico（以前称为粘多醣）。它是由分解硫酸角质素和硫酸软骨素的酶 N-乙酰半乳糖胺-6-硫酸酯酶 (GALNS) 或 β-半乳糖苷酶缺乏引起的。当这些糖分在细胞、组织和器官中积聚时，会导致骨骼缺陷、身材矮小、关节异常、呼吸道阻塞等呼吸系统疾病、心臟病、听力损失和角膜混浊。 MPS-IV 无法治疗方法，治疗方法仅限于症状治疗。然而，酵素替代疗法（ERT）和造血干细胞移植（HSCT）在控制症状和改善患者生活品质方面已取得一些成功。 MPS IV 是一种遗传性疾病。这意味着它通常在家庭内部传承。如果父母双方都携带无功能的 MPS IV 基因，则每个孩子患有 MPS IV 的几率为 25%（四分之一）。这被称为体染色体隐性遗传。

市场动态：

全球莫基奥症候群 (MPS-IV) 药物市场主要受到这种罕见遗传病患疾病率上升、对可用治疗方案的认识不断提高以及具有新作用机制的药物的强大管道的推动。然而，罕见疾病的高治疗成本以及生产重组酶的挑战正在抑制市场的成长。儘管如此，治疗方法的改进、罕见疾病研发的增加以及提供市场独占权的孤儿药资格的认定，为该市场的关键参与者提供了有利可图的机会。

本研究的主要特点

• 本报告对全球莫尔基奥症候群 (MPS-IV) 药物市场进行了详细分析，并以 2024 年为基准年，给出了预测期 (2025-2032) 的市场规模和年复合成长率(CAGR)。
• 它还强调了各个领域的潜在商机，并说明了该市场的有吸引力的投资提案矩阵。
• 它还提供了有关市场驱动因素、限制因素、机会、新产品发布和核准、市场趋势、区域前景和主要企业采用的竞争策略的重要见解。
• 主要公司简介包括 BioMarin Pharmaceutical Inc.、工业有限公司、辉瑞公司、赛诺菲、诺和诺德 A/S、Concert Pharmaceuticals, Inc.、Alexion Pharmaceuticals Inc.、诺华公司、安进公司、Sangamo Therapeutics 和 JCR Pharmaceuticals Co.。
• 本报告的见解将使负责人和企业经营团队能够就未来的产品发布、高端市场策略、市场扩张和行销策略做出明智的决策。
• 全球莫基奥症候群（MPS-IV）药物市场报告迎合了该行业的各个相关人员，例如投资者、供应商、产品製造商、经销商、新进业者和金融分析师。
• 相关人员可以透过用于分析全球莫尔基奥症候群（MPS-IV）药物市场的各种策略矩阵来促进决策。

目录

第一章 调查目的与前提条件

• 研究目标
• 先决条件
• 简称

第二章 市场展望

• 报告描述
  • 市场定义和范围
• 执行摘要
• Coherent Opportunity Map（COM）

第三章市场动态、法规与趋势分析

• 市场动态
  • 加强宣传宣传活动
  • 缺乏熟练的医疗保健专业人员
  • 医疗成本上涨
• 主要亮点
• 监管情景
• 近期动态
• PEST分析
• 波特分析
• 产品发布
• 合併、收购和合作

4. 全球莫基奥症候群（MPS-IV）药物市场-冠状病毒（COVID-19）大流行的影响

• COVID-19流行病学
• 供需侧分析
• 经济影响

5. 2020 年至 2032 年全球莫基奥症候群（MPS-IV）药物市场（依产品类型）

• 2025年和2032年市场占有率分析（％）
• 与前一年同期比较成长分析
• 细分趋势
• 固态剂型
• 市场规模及预测、与前一年同期比较
• 锭剂
• 胶囊
• 液体剂型
• 注射
• 其他的

6. 全球莫基奥症候群（MPS-IV）药物市场，依治疗方法，2020 年至 2032 年

• 2025年和2032年市场占有率分析（％）
• 与前一年同期比较成长分析
• 细分趋势
• 酵素替代疗法
• 造血干细胞治疗
• 基因治疗
• 其他的

7. 2020 年至 2032 年全球 Morquio 症候群（MPS-IV）药物市场（依疾病类型）

• 2025年和2032年市场占有率分析（％）
• 与前一年同期比较成长分析
• 细分趋势
• 莫尔基奥
• 莫尔基奥

8. 全球莫基奥症候群（MPS-IV）药物市场（依分销管道划分），2020 年至 2032 年

• 2025年和2032年市场占有率分析（％）
• 与前一年同期比较成长分析
• 细分趋势
• 医院药房
• 网路药局
• 零售药局

9. 2020 年至 2032 年全球莫基奥症候群（MPS-IV）药物市场（按地区）

• 2025年及2032年区域市场占有率分析（％）
• 与前一年同期比较
• 国家趋势
• 北美洲
• 美国
• 加拿大
• 欧洲
• 德国
• 英国
• 西班牙
• 法国
• 义大利
• 俄罗斯
• 其他欧洲国家
• 亚太地区
• 中国
• 印度
• 日本
• 澳洲
• 韩国
• ASEAN
• 其他亚太地区
• 拉丁美洲
• 巴西
• 阿根廷
• 墨西哥
• 其他拉丁美洲
• 中东
• 海湾合作委员会国家
• 以色列
• 其他中东地区
• 非洲
• 北非
• 中部非洲
• 南非

第十章 竞争格局

• BioMarin Pharmaceutical Inc.
• Takeda Pharmaceutical Company Limited.
• Pfizer Inc.
• Sanofi
• Novo Nordisk A/S
• Concert Pharmaceuticals, Inc.
• Alexion Pharmaceuticals Inc.
• Novartis AG
• Amgen Inc.
• Sangamo Therapeutics
• JCR Pharmaceuticals Co., Ltd.

第 11 章 章节

• 参考
• 调查方法
• 关于出版商

Global Morquio Syndrome (MPS-IV) Drug Market is estimated to be valued at USD 1.9 Mn in 2025 and is expected to reach USD 4.4 Mn by 2032, growing at a compound annual growth rate (CAGR) of 12.7% from 2025 to 2032.

Figure. Global Morquio Syndrome (MPS-IV) Drug Market Share (%), By Region 2025

Mucopolysaccharidosis type IV (MPS IV) is a rare disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (formerly called mucopolysaccharides). It is caused by a deficiency in the enzymes N-acetylgalactosamine-6-sulfatase (GALNS) or beta-galactosidase which is responsible for breaking down keratan sulfate and chondroitin sulfate. The accumulation of these sugars in cells, tissues, and organs can lead to skeletal dysplasia, short stature, joint abnormalities, and respiratory issues like airway obstruction, heart defects, hearing loss, and corneal opacity. There is no cure for MPS-IV and the treatment options are limited to symptom management. However, enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) have shown some success in management of symptoms and improvement in quality of life for patients. MPS IV is an inherited disorder. This means, it is typically passed down through families. If both parents carry a nonworking copy of a gene that is related to this condition, each of their children has a 25% (1 in 4) chance of developing the disease. This is called an autosomal recessive trait.

Market Dynamics:

Global morquio syndrome (MPS-IV) drug market is primarily driven by the rising prevalence of this rare genetic disorder, growing awareness about available treatment options and strong pipeline drugs with novel mechanisms of action. However, high cost of therapy due to rarity of disease as well as challenges that are associated with production of recombinant enzymes are some of the factors restraining the market growth. Nonetheless, development of improved treatment modalities, increasing research funding for rare diseases and orphan drug designation providing market exclusivity offers lucrative opportunities for the key market players in this market.

Key features of the study:

• This report provides in-depth analysis of the global morquio syndrome (MPS-IV) drug market, and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2025-2032), considering 2024 as the base year
• It elucidates potential revenue 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 morquio syndrome (MPS-IV) drug 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 BioMarin Pharmaceutical Inc., Takeda Pharmaceutical Company Limited., Pfizer Inc., Sanofi, Novo Nordisk A/S, Concert Pharmaceuticals, Inc., Alexion Pharmaceuticals Inc., Novartis AG, Amgen Inc., Sangamo Therapeutics, and JCR Pharmaceuticals Co., Ltd.
• 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 morquio syndrome (MPS-IV) drug 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 morquio syndrome (MPS-IV) drug market

Detailed Segmentation:

• Global Morquio Syndrome (MPS-IV) Drug Market, By Product Type
  • Solid Dosage Form
  • Tablets
  • Capsules
  • Liquid Dosage Form
  • Injectable
  • Others
• Global Morquio Syndrome (MPS-IV) Drug Market, By Treatment
  • Enzyme Replacement Therapy
  • Hematopoietic Stem Cell Therapy
  • Gene Therapy
  • Others
• Global Morquio Syndrome (MPS-IV) Drug Market, By Disease Type
  • Morquio A
  • Morquio B
• Global Morquio Syndrome (MPS-IV) Drug Market, By Distribution Channel
  • Hospital Pharmacies
  • Online Pharmacies
  • Retail Pharmacies
• Global Morquio Syndrome (MPS-IV) Drug Market, By Region
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East & Africa
• Company Profiles
  • BioMarin Pharmaceutical Inc.
  • Takeda Pharmaceutical Company Limited.
  • Pfizer Inc.
  • Sanofi
  • Novo Nordisk A/S
  • Concert Pharmaceuticals, Inc.
  • Alexion Pharmaceuticals Inc.
  • Novartis AG
  • Amgen Inc.
  • Sangamo Therapeutics
  • JCR Pharmaceuticals Co., Ltd.

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 Product Type
  • Market Snippet, By Treatment
  • Market Snippet, By Disease Type
  • Market Snippet, By Distribution Channel
  • Market Snippet, By Region
• Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

• Market Dynamics
  • Increasing Launch of Awareness Campaigns
  • Lack of skilled healthcare professionals
  • Rising healthcare expenditure
• Key Highlights
• Regulatory Scenario
• Recent Trends
• PEST Analysis
• PORTER's Analysis
• Product Launches
• Mergers, Acquisitions, and Collaborations

4. Global Morquio Syndrome (MPS-IV) Drug Market - Impact of Coronavirus (COVID-19) Pandemic

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

5. Global Morquio Syndrome (MPS-IV) Drug Market, By Product Type, 2020-2032, (US$ Mn)

• Introduction
• Market Share Analysis, 2025 and 2032 (%)
• Y-o-Y Growth Analysis, 2021 - 2032
• Segment Trends
• Solid Dosage Form
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Tablets
• Capsules
• Liquid Dosage Form
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Injectable
• Others

6. Global Morquio Syndrome (MPS-IV) Drug Market, By Treatment, 2020-2032, (US$ Mn)

• Introduction
• Market Share Analysis, 2025 and 2032 (%)
• Y-o-Y Growth Analysis, 2021 - 2032
• Segment Trends
• Enzyme Replacement Therapy
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Hematopoietic Stem Cell Therapy
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Gene Therapy
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Others
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)

7. Global Morquio Syndrome (MPS-IV) Drug Market, By Disease Type, 2020-2032, (US$ Mn)

• Introduction
• Market Share Analysis, 2025 and 2032 (%)
• Y-o-Y Growth Analysis, 2021 - 2032
• Segment Trends
• Morquio A
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Morquio B
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)

8. Global Morquio Syndrome (MPS-IV) Drug Market, By Distribution Channel, 2020-2032, (US$ Mn)

• Introduction
• Market Share Analysis, 2025 and 2032 (%)
• Y-o-Y Growth Analysis, 2021 - 2032
• Segment Trends
• Hospital Pharmacies
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Online Pharmacies
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
• Retail Pharmacies
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)

9. Global Morquio Syndrome (MPS-IV) Drug Market, By Region, 2020-2032, (US$ Mn)

• Introduction
• Market Share Analysis, By Region, 2025 and 2032 (%)
• Y-o-Y Growth Analysis, For Region, 2021 -2032
• Country Trends
• North America
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2020-2032,(US$
• Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
• U.S.
• Canada
• Europe
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2020-2032,(US$
• Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(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 Product Type, 2020-2032,(US$
• Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(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 Product Type, 2020-2032,(US$
• Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
• Brazil
• Argentina
• Mexico
• Rest of Latin America
• Middle East
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2020-2032,(US$
• Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
• GCC Countries
• Israel
• Rest of Middle East
• Africa
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2020-2032,(US$
• Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
• Market Size and Forecast, and Y-o-Y Growth, By Country/Region, 2020-2032,(US$ Mn)
• North Africa
• Central Africa
• South Africa

10. Competitive Landscape

• Company Profile
• BioMarin Pharmaceutical Inc.
• Company Highlights
• Product Portfolio
• Key Highlights
• Financial Performance
• Strategies
• Takeda Pharmaceutical Company Limited.
• Pfizer Inc.
• Sanofi
• Novo Nordisk A/S
• Concert Pharmaceuticals, Inc.
• Alexion Pharmaceuticals Inc.
• Novartis AG
• Amgen Inc.
• Sangamo Therapeutics
• JCR Pharmaceuticals Co., Ltd.
• Analyst Views

11. Section

• References
• Research Methodology
• About us