The treatment of Mucopolysaccharidosis (MPS) is primarily focused on managing symptoms, improving the quality of life, and slowing down the progression of the disease. MPS is a group of rare genetic disorders characterized by the accumulation of glycosaminoglycans (GAGs) in cells and tissues due to deficiencies in specific enzymes responsible for breaking down these complex sugars. Enzyme Replacement Therapy (ERT): This is the mainstay treatment for many types of MPS. In ERT, the missing or deficient enzyme is administered intravenously to help break down the accumulated GAGs. Although ERT can be beneficial, it may not effectively treat neurological symptoms because the enzyme may not efficiently cross the blood-brain barrier. Hematopoietic Stem Cell Transplantation (HSCT): Also known as bone marrow transplantation, HSCT involves transplanting healthy stem cells from a donor into the patient's bone marrow. The donor cells can produce the missing enzyme, leading to a potential reduction in GAG accumulation. HSCT is most effective when performed early in the disease course before significant irreversible damage has occurred.

 Market Dynamics     

Market players are focusing on various inorganic growth strategies such as collaborations for the treatment mucopolysaccharidosis (MPS), which is expected to boost the global mucopolysaccharidosis (MPS) treatment market growth. For instance, In September 2021, Takeda Pharmaceuticals, a patient-focused, R&D-driven, global biopharmaceutical company committed to bringing Better Health and a Brighter Future, entered into an exclusive collaboration and license agreement with JCR Pharmaceuticals is a global specialty pharmaceuticals company, to commercialize JR-141, an investigational next-generation recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase (IDS) enzyme for the treatment of Hunter syndrome. Under the terms of the agreement, Takeda commercialized JR-141 outside of the U.S., including Canada, Europe, and other regions (excluding Japan and certain other Asia Pacific countries). JCR received an upfront payment for such an ex-US license and is eligible to receive additional development and commercial milestones, as well as tiered royalties on potential sales.

Key features of the study:

• This report provides an in-depth analysis of the global mucopolysaccharidosis (MPS) treatment market and provides market size (US$ Billion) and compound annual growth rate (CAGR) for the forecast period (2023–2030), considering 2022 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 mucopolysaccharidosis (MPS) 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 Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Abeona Therapeutics Inc., Eloxx Pharmaceuticals, Esteve, Immusoft Corporation, Inventiva, GC Pharma, JCR Pharmaceuticals Co Ltd., RegenxBio Inc., Sangamo Therapeutics, Inc., Bioasis Technologies Inc., and Paradigm Biopharmaceuticals Ltd.
• Global mucopolysaccharidosis (MPS) 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 mucopolysaccharidosis (MPS) treatment market.

Detailed Segmentation:

• Global Mucopolysaccharidosis (MPS) Treatment Market, By Treatment:
  • Enzyme Replacement Therapy
  • Stem Cell Therapy
• Global Mucopolysaccharidosis (MPS) Treatment Market, By MPS Type:
  • MPS-I
  • MPS-II
  • MPS-IV
  • MPS-VI
  • MPS-VII
  • Others (MPS-III and MPS-IX)
• Global Mucopolysaccharidosis (MPS) Treatment Market, By End User:
  • Hospitals
  • Specialty Clinics
  • Others
• Global Mucopolysaccharidosis (MPS) Treatment, By Region:
  • North America
    
    • U.S.
    • Canada
  • Latin America
    
    • Brazil
    • Mexico
    • Argentina
    • Rest of Latin America
  • Europe
    
    • Germany
    • U.K.
    • France
    • Italy
    • Spain
    • Russia
    • Rest of Europe
  • Asia Pacific
    
    • China
    • India
    • Japan
    • Australia
    • South Korea
    • ASEAN
    • Rest of Asia Pacific
  • Middle East
    
    • GCC
    • Israel
    • Rest of Middle East
  • Africa
    
    • South Africa
    • Central Africa
    • North Africa
• Company Profiles
  • Takeda Pharmaceutical Company Limited
  • BioMarin Pharmaceuticals, Inc.
  • Ultragenyx Pharmaceutical Inc.
  • Sarepta Therapeutics
  • Abeona Therapeutics Inc.
  • Eloxx Pharmaceuticals
  • Esteve
  • Immusoft Corporation
  • Inventiva
  • GC Pharma
  • JCR Pharmaceuticals Co Ltd.
  • RegenxBio Inc.
  • Sangamo Therapeutics, Inc.
  • Bioasis Technologies Inc.
  • Paradigm Biopharmaceuticals Ltd.