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Pompe Disease Treatment Market Size - By Treatment Type (ERT, Supportive Care), By Disease Type (Infantile, Juvenile), By Therapy Type (Monotherapy, Combination Therapy), By End-user (Hospitals, Infusion Care) & Forecast, 2024 - 2032

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  • Abeona Therapeutics Inc.
  • Amicus Therapeutics, Inc.
  • Aro Biotherapeutics Company
  • Audentes Therapeutics(Astellas Pharma Inc.)
  • Bayer AG
  • F. Hoffmann-La Roche Ltd.
  • Genethon
  • Maze Therapeutics
  • M6P Therapeutics
  • Oxyrane
  • Sanofi
  • Valerion Therapeutics
ksm 24.05.02

Global Pompe Disease Treatment Market size will witness a 4.4% CAGR between 2024 and 2032, propelled by escalating investments and funding in treatment initiatives. As awareness about Pompe disease grows and its impact on affected individuals becomes better understood, there has been a concerted effort to develop effective therapies. For instance, in November 2023, in a Series B financing round, clinical-stage biotechnology firm Aro Biotherapeutics secured $41.5 million to further progress the development of its primary candidate, ABX1100, targeting Pompe disease.

Pharmaceutical companies, research institutions, and government organizations are channeling resources into research and development projects aimed at discovering novel treatments and improving existing therapies for Pompe disease. Additionally, initiatives such as patient advocacy groups and public-private partnerships further drive investments in treatment development. This collective effort helps expand the treatment options available to Pompe disease patients while underscoring the importance of collaborative approaches in addressing rare diseases and improving patient outcomes.

The overall Pompe Disease Treatment Industry is classified based on the treatment type, disease type, therapy type, end-use, and region.

The supportive care segment will register a significant growth rate by 2032. As Pompe disease affects multiple organs and systems in the body, patients often require comprehensive supportive care to manage symptoms and improve their quality of life. This includes interventions such as respiratory support, physical therapy, and nutritional management. As healthcare providers prioritize holistic approaches to patient care, the demand for supportive therapies in the Pompe Disease Treatment Market continues to grow, ensuring comprehensive management and improved outcomes for affected individuals.

The Pompe disease treatment market from infantile segment will exhibit significant growth from 2023 to 2032. Infantile-onset Pompe disease, a severe form of the disorder, requires prompt and comprehensive treatment to mitigate symptoms and improve outcomes. Therapies such as enzyme replacement therapy (ERT) and supportive care play a crucial role in managing infantile Pompe disease. As early diagnosis and intervention are pivotal for infants, there is a heightened demand for effective treatments tailored to address the specific needs of this vulnerable population, driving innovation and advancements in the Pompe Disease Treatment Market.

Europe Pompe disease treatment market will showcase a commendable CAGR from 2024 to 2032. With advanced healthcare systems and increasing awareness about rare diseases, there's a growing emphasis on early diagnosis and effective management of Pompe disease. European countries prioritize access to innovative therapies, including enzyme replacement therapy (ERT) and supportive care, to improve patient outcomes.

As healthcare providers and policymakers collaborate to address the needs of Pompe disease patients, the demand for comprehensive treatment options in Europe continues to drive market growth and foster advancements in healthcare delivery. For instance, in February 2024, Sanofi, the French biopharmaceutical company, reported encouraging outcomes for its enzyme replacement therapy, avalglucosidase alfa, in diverse Pompe disease patient cohorts.

Table of Contents

Chapter 1 Methodology & Scope

  • 1.1 Market scope & definitions
  • 1.2 Base estimates & calculations
  • 1.3 Data validation
  • 1.4 Forecast parameters
  • 1.5 Data sources
    • 1.5.1 Primary
    • 1.5.2 Secondary
      • 1.5.2.1 Paid sources
      • 1.5.2.2 Public sources

Chapter 2 Executive Summary

  • 2.1 Industry 360 degree synopsis

Chapter 3 Industry Insights

  • 3.1 Industry ecosystem analysis
  • 3.2 Industry impact forces
    • 3.2.1 Growth drivers
      • 3.2.1.1 Growing incidence of pompe disease
      • 3.2.1.2 Expanding R&D efforts
      • 3.2.1.3 Growing regulatory support
      • 3.2.1.4 Increasing emphasis on early diagnosis advancement
      • 3.2.1.5 Favorable reimbursement policies
    • 3.2.2 Industry pitfalls & challenges
      • 3.2.2.1 Limited treatment options
      • 3.2.2.2 High treatment costs
  • 3.3 Growth potential analysis
  • 3.4 Pipeline assessment
  • 3.5 Reimbursement scenario
  • 3.6 Regulatory landscape
  • 3.7 Porter's analysis
    • 3.7.1 Supplier power
    • 3.7.2 Buyer power
    • 3.7.3 Threat of new entrants
    • 3.7.4 Threat of substitutes
    • 3.7.5 Industry rivalry
  • 3.8 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

  • 4.1 Introduction
  • 4.2 Company matrix analysis
  • 4.3 Company market share analysis
  • 4.4 Competitive positioning matrix
  • 4.5 Strategic dashboard

Chapter 5 Market Estimates and Forecast, By Treatment Type, 2018 - 2032 ($ Mn)

  • 5.1 Key trends
  • 5.2 Enzyme replacement therapy (ERT)
  • 5.3 Supportive care

Chapter 6 Market Estimates and Forecast, By Disease Type, 2018 - 2032 ($ Mn)

  • 6.1 Key trends
  • 6.2 Infantile
  • 6.3 Juvenile

Chapter 7 Market Estimates and Forecast, By Therapy, 2018 - 2032 ($ Mn)

  • 7.1 Key trends
  • 7.2 Monotherapy
  • 7.3 Combination therapy

Chapter 8 Market Estimates and Forecast, By End-User, 2018 - 2032 ($ Mn)

  • 8.1 Key trends
  • 8.2 Hospitals
  • 8.3 Infusion centers
  • 8.4 Other end-users

Chapter 9 Market Estimates and Forecast, By Region, 2018 - 2032 ($ Mn)

  • 9.1 Key trends, by region
  • 9.2 North America
    • 9.2.1 U.S.
    • 9.2.2 Canada
  • 9.3 Europe
    • 9.3.1 Germany
    • 9.3.2 UK
    • 9.3.3 France
    • 9.3.4 Spain
    • 9.3.5 Italy
    • 9.3.6 Rest of Europe
  • 9.4 Asia Pacific
    • 9.4.1 Japan
    • 9.4.2 China
    • 9.4.3 India
    • 9.4.4 Australia
    • 9.4.5 Rest of Asia Pacific
  • 9.5 Latin America
    • 9.5.1 Brazil
    • 9.5.2 Mexico
    • 9.5.3 Rest of Latin America
  • 9.6 Middle East and Africa
    • 9.6.1 South Africa
    • 9.6.2 Saudi Arabia
    • 9.6.3 Rest of Middle East and Africa

Chapter 10 Company Profiles

  • 10.1 Abeona Therapeutics Inc.
  • 10.2 Amicus Therapeutics, Inc.
  • 10.3 Aro Biotherapeutics Company
  • 10.4 Audentes Therapeutics (Astellas Pharma Inc.)
  • 10.5 Bayer AG
  • 10.6 F. Hoffmann-La Roche Ltd.
  • 10.7 Genethon
  • 10.8 Maze Therapeutics
  • 10.9 M6P Therapeutics
  • 10.10 Oxyrane
  • 10.11 Sanofi
  • 10.12 Valerion Therapeutics
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