Mucopolysaccharidosis (MPS) Treatment Market

By Treatment;

Enzyme Replacement Therapy and Stem Cell Therapy

By MPS;

MPS-I, MPS-II, MPS-IV, MPS-VI, MPS-VII, and Others

By End-User;

Hospitals, Pharmaceuticals, and Research Institutions

By Geography;

North America, Europe, Asia Pacific, Middle East & Africa, and Latin America - Report Timeline (2021 - 2031)
Report ID: Rn758292788 Published Date: June, 2025 Updated Date: July, 2025

Mucopolysaccharidosis (MPS) Treatment Market Overview

Mucopolysaccharidosis (MPS) Treatment Market (USD Million)

Mucopolysaccharidosis (MPS) Treatment Market was valued at USD 2,191.13 million in the year 2024. The size of this market is expected to increase to USD 4,062.40 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 9.2%.


Mucopolysaccharidosis (MPS) Treatment Market

*Market size in USD million

CAGR 9.2 %


Study Period2025 - 2031
Base Year2024
CAGR (%)9.2 %
Market Size (2024)USD 2,191.13 Million
Market Size (2031)USD 4,062.40 Million
Market ConcentrationMedium
Report Pages379
2,191.13
2024
4,062.40
2031

Major Players

  • Shire (now part of Takeda Pharmaceutical Company Limited)
  • Sanofi Genzyme
  • BioMarin Pharmaceutical Inc.
  • Ultragenyx Pharmaceutical Inc.
  • Sangamo Therapeutics, Inc.
  • Denali Therapeutics Inc.
  • ArmaGen Inc.
  • REGENXBIO Inc.
  • JCR Pharmaceuticals Co., Ltd.
  • Abeona Therapeutics Inc.
  • Lysogene S.A.
  • Orchard Therapeutics plc
  • Homology Medicines, Inc.
  • Avrobio, Inc.
  • Phoenix Tissue Repair, Inc.

Market Concentration

Consolidated - Market dominated by 1 - 5 major players

Mucopolysaccharidosis (MPS) Treatment Market

Fragmented - Highly competitive market without dominant players


The Mucopolysaccharidosis (MPS) Treatment Market is experiencing considerable growth due to rising awareness and enhanced diagnostic capabilities. The adoption of enzyme replacement therapies has increased by over 65%, driving the need for innovative treatment options. A surge in early detection rates has prompted a shift toward targeted therapeutic strategies, enabling better clinical outcomes. This progression is fostering both opportunities and expansion within the treatment landscape.

Technological Advancements Driving Innovation
Advanced research in gene therapy and biopharmaceuticals has influenced the market, with over 58% of treatments now incorporating next-generation solutions. These technological advancements have enabled the development of more precise and effective drug formulations. Continuous innovation in delivery mechanisms is also contributing to improved patient compliance. The integration of digital health platforms is becoming a core strategy to support treatment personalization and efficiency.

Strategic Partnerships and Collaborations
Over 60% of key stakeholders are engaging in active collaborations and partnerships to accelerate therapeutic development. These joint ventures are focused on scaling up clinical trials and enhancing the production of biopharmaceutical drugs. Strategic mergers among biotech firms are strengthening market penetration and facilitating a broader treatment portfolio. This collaborative ecosystem is essential for addressing rare disease complexities in MPS.

Focus on Personalized Therapeutic Strategies
The emphasis on personalized medicine is gaining momentum, with more than 55% of treatment models shifting toward individualized protocols. These strategies involve the integration of genetic profiling and tailored therapy, providing a significant boost to treatment precision. This shift is expected to elevate patient outcomes and optimize healthcare resources. Ongoing research supports the trend, underlining a proactive and customized approach to care.

  1. Introduction
    1. Research Objectives and Assumptions
    2. Research Methodology
    3. Abbreviations
  2. Market Definition & Study Scope
  3. Executive Summary
    1. Market Snapshot, By Treatment
    2. Market Snapshot, By MPS
    3. Market Snapshot, By End-user
    4. Market Snapshot, By Region
  4. Mucopolysaccharidosis (MPS) Treatment Market Dynamics
    1. Drivers, Restraints and Opportunities
      1. Drivers
        1. Increasing MPS awareness and diagnosis
        2. Biotech and genetics advances
        3. Pharma R&D investments
        4. Favorable orphan drug regulations
        5. Newborn screening expansion
      2. Restraints
        1. Diagnosis and subtype challenges
        2. Limited specialist access
        3. Treatment side effects
        4. Regional treatment guideline variance
        5. Disease complexity and variability
      3. Opportunities
        1. Gene therapy breakthroughs
        2. Newborn screening program expansion
        3. Precision medicine adoption
        4. Data-sharing initiatives
        5. Public-private rare disease partnerships
    2. PEST Analysis
      1. Political Analysis
      2. Economic Analysis
      3. Social Analysis
      4. Technological Analysis
    3. Porter's Analysis
      1. Bargaining Power of Suppliers
      2. Bargaining Power of Buyers
      3. Threat of Substitutes
      4. Threat of New Entrants
      5. Competitive Rivalry
  5. Market Segmentation
    1. Mucopolysaccharidosis (MPS) Treatment Market, By Treatment, 2021 - 2031 (USD Million)
      1. Enzyme Replacement Therapy
      2. Stem Cell Therapy
    2. Mucopolysaccharidosis (MPS) Treatment Market,By MPS, 2021 - 2031 (USD Million)
      1. MPS-I
      2. MPS-II
      3. MPS-IV
      4. MPS-VI
      5. MPS-VII
      6. Others
    3. Mucopolysaccharidosis (MPS) Treatment Market, By End-User, 2021 - 2031 (USD Million)
      1. Hospitals,
      2. Pharmaceuticals
      3. Research Institutions
    4. Mucopolysaccharidosis (MPS) Treatment Market, By Geography, 2021 - 2031 (USD Million)
      1. North America
        1. United States
        2. Canada
      2. Europe
        1. Germany
        2. United Kingdom
        3. France
        4. Italy
        5. Spain
        6. Nordic
        7. Benelux
        8. Rest of Europe
      3. Asia Pacific
        1. Japan
        2. China
        3. India
        4. Australia & New Zealand
        5. South Korea
        6. ASEAN (Association of South East Asian Countries)
        7. Rest of Asia Pacific
      4. Middle East & Africa
        1. GCC
        2. Israel
        3. South Africa
        4. Rest of Middle East & Africa
      5. Latin America
        1. Brazil
        2. Mexico
        3. Argentina
        4. Rest of Latin America
  6. Competitive Landscape
    1. Company Profiles
      1. Shire (now part of Takeda Pharmaceutical Company Limited)
      2. Sanofi Genzyme
      3. BioMarin Pharmaceutical Inc.
      4. Ultragenyx Pharmaceutical Inc.
      5. Sangamo Therapeutics, Inc.
      6. Denali Therapeutics Inc.
      7. ArmaGen Inc.
      8. REGENXBIO Inc.
      9. JCR Pharmaceuticals Co., Ltd.
      10. Abeona Therapeutics Inc.
      11. Lysogene S.A.
      12. Orchard Therapeutics plc
      13. Homology Medicines, Inc.
      14. Avrobio, Inc.
      15. Phoenix Tissue Repair, Inc.
  7. Analyst Views
  8. Future Outlook of the Market