Mucopolysaccharidosis (MPS) Treatment Market Size & Share, by Type (Hurler syndrome, Hunter syndrome); End-user (Hospitals, Pharmaceuticals, Research Institutions) - Global Supply & Demand Analysis, Growth Forecasts, Statistics Report 2023-2035

  • Report ID: 5215
  • Published Date: Sep 04, 2023
  • Report Format: PDF, PPT

Global Market Size, Forecast, and Trend Highlights Over 2023 - 2035

Mucopolysaccharidosis Treatment Market size is anticipated to reach USD 20 billion by the end of 2035, growing at a CAGR of 8% during the forecast period, i.e., 2023 – 2035. In the year 2022, the industry size of mucopolysaccharidosis treatment was over USD 10 billion. HSCT, also known as bone marrow transplant, involves replacing the patient's faulty bone marrow or stem cells with healthy donor cells that produce the missing enzyme. In addition to these established treatments, ongoing research and development efforts were focused on finding new therapies and improving existing ones. These efforts might include gene therapy, small molecule drugs, and other innovative approaches aimed at addressing the underlying genetic and biochemical factors contributing to MPS.

Mucopolysaccharidosis (MPS) refers to a group of rare genetic disorders known as lysosomal storage disorders. These disorders are characterized by the accumulation of glycosaminoglycans (GAGs) due to the deficiency of specific enzymes required for their breakdown within lysosomes. This leads to a range of symptoms and complications affecting various organs and systems in the body.

Mucopolysaccharidosis (MPS) Treatment Market
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Mucopolysaccharidosis (MPS) Treatment Market: Growth Drivers and Challenges

Growth Drivers

  • Advancements in Enzyme Replacement Therapy (ERT): Enzyme Replacement Therapy (ERT) stands as a cornerstone in the network management solutions of MPS, offering targeted intervention by providing the deficient enzymes responsible for GAG metabolism. The sector has experienced substantial growth, with research continually refining the efficacy and delivery methods of ERT. According to a study, ERT has demonstrated considerable improvements in clinical outcomes, with patients experiencing enhanced pulmonary function and reduction in hepatosplenomegaly.
  • Increased Awareness and Early Diagnosis: The growing awareness about MPS among healthcare professionals, caregivers, and the general public has led to early diagnosis and intervention, a critical factor in improving patient outcomes. Timely diagnosis empowers healthcare providers to initiate treatments during the earlier stages of the disease.
  • Emergence of Novel Therapies: The MPS treatment landscape has expanded beyond traditional approaches, with the emergence of novel therapies like gene therapy and substrate reduction therapy. These innovative modalities hold promise in addressing the genetic basis of MPS and minimizing GAG accumulation.


  • Limited Awareness and Early Diagnosis: Despite efforts to raise awareness, many cases of MPS still go undiagnosed or are diagnosed at a later stage. This delay in diagnosis can impede the effectiveness of treatments, as interventions are often most impactful when initiated early in the disease course.
  • Complexity of Disease Subtypes
  • Access to Specialized Care

Mucopolysaccharidosis (MPS) Treatment: Key Insights

Base Year


Forecast Year




Base Year Market Size (2022)

~ USD 10 Billion

Forecast Year Market Size (2035)

~ USD 20 Billion

Regional Scope

  • North America (U.S., and Canada)
  • Latin America (Mexico, Argentina, Rest of Latin America)
  • Asia-Pacific (Japan, China, India, Indonesia, Malaysia, Australia, Rest of Asia-Pacific)
  • Europe (U.K., Germany, France, Italy, Spain, Russia, NORDIC, Rest of Europe)
  • Middle East and Africa (Israel, GCC North Africa, South Africa, Rest of Middle East and Africa)
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Mucopolysaccharidosis (MPS) Treatment Segmentation

Type (Hurler syndrome, Hunter syndrome)

The hunter syndrome segment is estimated to hold 60% share of the mucopolysaccharidosis treatment market in the year 2035. Enzyme Replacement Therapy (ERT) has revolutionized the treatment of Hunter syndrome, offering patients an opportunity for improved quality of life by addressing the underlying enzyme deficiency. Advances in ERT methodologies and formulations have enhanced its efficacy and accessibility. A study reported that ERT with idursulfase led to substantial reductions in urinary glycosaminoglycan (GAG) levels and improvements in various clinical parameters in Hunter syndrome patients.

End User (Hospitals, Pharmaceuticals, Research Institutions)

Mucopolysaccharidosis (MPS) treatment market from the hospitals segment is expected to garner a significant share in the year 2035. Increasing awareness among healthcare professionals and the public about MPS has led to higher rates of diagnosis. This trend has driven a surge in patients seeking specialized care within hospital settings, contributing to the growth of the hospitals segment. The Global Genes organization reports that greater awareness campaigns have resulted in an upswing in MPS diagnoses, highlighting the importance of healthcare facilities in providing accurate diagnoses.

Our in-depth analysis of the global mucopolysaccharidosis treatment market includes the following segments:


  • Hurler syndrome
  • Hunter syndrome



           End User

  • Hospitals
  • Pharmaceuticals
  • Research Institutions

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MPS Treatment Market – Regional Synopsis

APAC Market Forecast

The mucopolysaccharidosis treatment market in the Asia Pacific region is projected to hold the largest market share of 38% by the end of 2035. Growing awareness campaigns and improved medical education have led to higher rates of MPS diagnoses in the APAC region. The resulting rise in patient identification has catalyzed the demand for effective treatment options. According to a report, efforts to increase disease awareness have resulted in a substantial increase in MPS diagnoses across various countries in the Asia Pacific region. For instance, MPS II (Hunter syndrome) cases diagnosed in China doubled over a five-year period.

North American Market Statistics

The mucopolysaccharidosis treatment market in the North America region is projected to hold the second largest share during the forecast period. North America's advanced healthcare infrastructure, including specialized treatment centers and expert medical professionals, contributes to improved MPS patient care, diagnosis, and access to innovative therapies. The Canadian Society for Mucopolysaccharide and Related Diseases (Canadian MPS Society) highlights the establishment of specialized treatment centers and the growth of expertise in managing MPS cases across North America.

Research Nester
Mucopolysaccharidosis (MPS) Treatment Market Size
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Companies Dominating the Mucopolysaccharidosis Treatment Market

    • Shire Inc.
      • Company Overview
      • Business Strategy
      • Key Product Offerings
      • Financial Performance
      • Key Performance Indicators
      • Risk Analysis
      • Recent Development
      • Regional Presence
      • SWOT Analysis
    • Sanofi Genzyme
    • BioMarin Pharmaceutical
    • Ultragenyx Pharmaceutical
    • Regenxbio
    • Abeona Therapeutics
    • Sangamo Therapeutics
    • Denali Therapeutics
    • Lysogene
    • ArmaGen

In the News

  • Beyfortus™ is a novel, long-acting RSV pre-exposure prophylaxis (pre-EP) monoclonal antibody developed by Sanofi and Genzyme. The vaccine was found to be safe and effective in preventing RSV infection in infants from 6 weeks to 5 months of age. The CDC's Advisory Committee on Immunization Practices (ACIP) voted unanimously to recommend routine use of Beyfortus™ for all infants in the United States.
  • Sanofi to acquire Qunol®, a fast-growing U.S. brand in the healthy aging segment. Qunol® is a leading brand of nicotinamide riboside (NR) supplements, which are naturally produced in the body and play a role in cellular energy production.

Author Credits:  Radhika Gupta, Shivam Bhutani

  • Report ID: 5215
  • Published Date: Sep 04, 2023
  • Report Format: PDF, PPT

Frequently Asked Questions (FAQ)

Increasing demand for healthcare sector is the major factor driving the market growth.

The market size of mucopolysaccharidosis (MPS) treatment is anticipated to attain a CAGR of 8% over the forecast period, i.e., 2023 – 2035.

The major players in the market are Shire (now part of Takeda Pharmaceutical Company), Sanofi Genzyme, BioMarin Pharmaceutical, Ultragenyx Pharmaceutical, Regenxbio, Abeona Therapeutics, and others.

The hospitals segment is anticipated to garner the largest market size by the end of 2035 and display significant growth opportunities.

The market in the Asia Pacific region is projected to hold the largest market share by the end of 2035 and provide more business opportunities in the future.
Mucopolysaccharidosis Treatment Market Report Scope

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