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Pompe Disease Treatment Market by Treatment (Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Chaperone-Advanced Replacement Therapy (CART), and Others), Route of Administration (Oral, Intravenous, and Others), Distribution Channel (Hospital and Clinics Pharmacies, Retail Pharmacies, Online Pharmacies, and Others), Indication Type (Infantile-Onset Pompe Disease (IOPD), Classic Infantile Form, Non-Classic Infantile Form, Late-Onset Pompe Disease (LOPD), and Others), and Region 2024-2032

January 2024 | 142 pages | ID: PF22D5CA2146EN
IMARC Group

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The global Pompe disease treatment market size reached US$ 1.0 Billion in 2023. Looking forward, IMARC Group expects the market to reach US$ 1.3 Billion by 2032, exhibiting a growth rate (CAGR) of 2.58% during 2024-2032. The increasing number of clinical studies and trials, the rising popularity of ERT, and the growing number of patients suffering from Pompe disease represent some of the key factors driving the market.

Pompe disease is a genetic disorder caused by the buildup of a complex sugar known as glycogen in the cells of the body, which affects organs, tissues, and muscles. It is an autosomal recessive glycogen storage syndrome caused by the deficiency of lysosomal enzyme acid a-glucosidase (GAA). It is associated with weak muscles, enlarged liver, trouble breathing, hearing problem, lung infection, irregular heartbeat, and losing weight. It is diagnosed using various tools and tests, including electromyography, X-rays, and magnetic resonance imaging (MRI). Pompe disease can be treated using medications, enzyme replacement therapy (ERT), substrate reduction therapy (SRT), chaperone-advanced replacement therapy (CART), and injections, which aid in improving the quality of life.

Pompe Disease Treatment Market Trends:
There is a considerable rise in the prevalence of genetic abnormalities among individuals and an increase in the number of patients suffering from Pompe disease. This, coupled with the growing Pompe disease among infants and increasing concerns among parents about the health of their newborns, represents one of the key factors bolstering the growth of the market. Moreover, the growing geriatric population that is susceptible to developing these medical disorders is resulting in an increasing demand for Pompe disease treatment. In addition, the expanding number of clinical studies and trials to develop new treatments for Pompe disease is influencing the market positively. Apart from this, the rising awareness among people about ERT for curing Pompe disease is favoring the market growth. Furthermore, there is an increase in awareness about various treatments, including stem cell transplant, bone marrow transplantation, chemotherapy, and immunotherapy, on account of their numerous advantages. This, along with the awareness about the easy availability of ERT treatment alternatives like lumizyme, is creating a positive outlook for the market. Besides this, there is a rise in the adoption of health insurance policies that provide financial assistance and minimize healthcare expenditure for Pompe disease treatment. Additionally, governments and non-governmental organizations (NGOs) of numerous countries are undertaking measures to offer quality healthcare facilities that are accessible to all. This, along with significant improvements in the diagnostic technologies, is anticipated to provide a favorable outlook to the market.

Key Market Segmentation:
IMARC Group provides an analysis of the key trends in each segment of the global Pompe disease treatment market, along with forecasts at the global, regional, and country level from 2024-2032. Our report has categorized the market based on treatment, route of administration, distribution channel, and indication type.

Treatment Insights:

Enzyme Replacement Therapy (ERT)
Substrate Reduction Therapy (SRT)
Chaperone-Advanced Replacement Therapy (CART)
Others

The report has provided a detailed breakup and analysis of the Pompe disease treatment market based on the treatment. This includes enzyme replacement therapy (ERT), substrate reduction therapy (SRT), chaperone-advanced replacement therapy (CART), and others. According to the report, enzyme replacement therapy (ERT) represented the largest segment.

Route of Administration Insights:

Oral
Intravenous
Others

A detailed breakup and analysis of the Pompe disease treatment market based on the route of administration has also been provided in the report. This includes oral, intravenous, and others.

Distribution Channel Insights:

Hospital and Clinics Pharmacies
Retail Pharmacies
Online Pharmacies
Others

A detailed breakup and analysis of the Pompe disease treatment market based on the distribution channel has also been provided in the report. This includes hospital and clinics, retail, and online pharmacies, and others. According to the report, hospital and clinics pharmacies accounted for the largest market share.

Indication Type Insights:

Infantile-Onset Pompe Disease (IOPD)
Classic Infantile Form
Non-Classic Infantile Form
Late-Onset Pompe Disease (LOPD)
Others

A detailed breakup and analysis of the Pompe disease treatment market based on the indication type has also been provided in the report. This includes infantile-onset Pompe disease (IOPD), classic infantile form, non-classic infantile form, late-onset Pompe disease (LOPD), and others. According to the report, late-onset Pompe disease (LOPD) accounted for the largest market share.

Regional Insights:

North America
United States
Canada
Asia Pacific
China
Japan
India
South Korea
Australia
Indonesia
Others
Europe
Germany
France
United Kingdom
Italy
Spain
Russia
Others
Latin America
Brazil
Mexico
Others
Middle East and Africa

The report has also provided a comprehensive analysis of all the major regional markets that include North America (the United States and Canada), Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others), Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others), Latin America (Brazil, Mexico, and others), and the Middle East and Africa. According to the report, North America was the largest market for Pompe disease treatment. Some of the factors driving the North America Pompe disease treatment market included the growing awareness about various alternative treatments, increasing number of clinical studies and trials, and well-established healthcare facilities.

Competitive Landscape:
The report has also provided a comprehensive analysis of the competitive landscape in the global Pompe disease treatment market. Detailed profiles of all major companies have also been provided. Some of the companies covered include Amicus Therapeutics Inc., Audentes Therapeutics Inc. (Astellas US Holding Inc.), Oxyrane UK Limited, Sanofi S.A., Spark Therapeutics Inc, etc.

Key Questions Answered in This Report:
How has the global Pompe disease treatment market performed so far and how will it perform in the coming years?
What are the drivers, restraints, and opportunities in the global Pompe disease treatment market?
What are the key regional markets?
Which countries represent the most attractive Pompe disease treatment markets?
What is the breakup of the market based on the treatment?
What is the breakup of the market based on the route of administration?
What is the breakup of the market based on the distribution channel?
What is the breakup of the market based on the indication type?
What is the competitive structure of the global Pompe disease treatment market?
Who are the key players/companies in the global Pompe disease treatment market?
1 PREFACE


2 SCOPE AND METHODOLOGY

2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
  2.3.1 Primary Sources
  2.3.2 Secondary Sources
2.4 Market Estimation
  2.4.1 Bottom-Up Approach
  2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 EXECUTIVE SUMMARY


4 INTRODUCTION

4.1 Overview
4.2 Key Industry Trends

5 GLOBAL POMPE DISEASE TREATMENT MARKET

5.1 Market Overview
5.2 Market Performance
5.3 Impact of COVID-19
5.4 Market Forecast

6 MARKET BREAKUP BY TREATMENT

6.1 Enzyme Replacement Therapy (ERT)
  6.1.1 Market Trends
  6.1.2 Market Forecast
6.2 Substrate Reduction Therapy (SRT)
  6.2.1 Market Trends
  6.2.2 Market Forecast
6.3 Chaperone-Advanced Replacement Therapy (CART)
  6.3.1 Market Trends
  6.3.2 Market Forecast
6.4 Others
  6.4.1 Market Trends
  6.4.2 Market Forecast

7 MARKET BREAKUP BY ROUTE OF ADMINISTRATION

7.1 Oral
  7.1.1 Market Trends
  7.1.2 Market Forecast
7.2 Intravenous
  7.2.1 Market Trends
  7.2.2 Market Forecast
7.3 Others
  7.3.1 Market Trends
  7.3.2 Market Forecast

8 MARKET BREAKUP BY DISTRIBUTION CHANNEL

8.1 Hospital & Clinics Pharmacies
  8.1.1 Market Trends
  8.1.2 Market Forecast
8.2 Retail Pharmacies
  8.2.1 Market Trends
  8.2.2 Market Forecast
8.3 Online Pharmacies
  8.3.1 Market Trends
  8.3.2 Market Forecast
8.4 Others
  8.4.1 Market Trends
  8.4.2 Market Forecast

9 MARKET BREAKUP BY INDICATION TYPE

9.1 Infantile-Onset Pompe Disease (IOPD)
  9.1.1 Market Trends
  9.1.2 Market Forecast
9.2 Classic Infantile Form
  9.2.1 Market Trends
  9.2.2 Market Forecast
9.3 Non-Classic Infantile Form
  9.3.1 Market Trends
  9.3.2 Market Forecast
9.4 Late-Onset Pompe Disease (LOPD)
  9.4.1 Market Trends
  9.4.2 Market Forecast
9.5 Others
  9.5.1 Market Trends
  9.5.2 Market Forecast

10 MARKET BREAKUP BY REGION

10.1 North America
  10.1.1 United States
    10.1.1.1 Market Trends
    10.1.1.2 Market Forecast
  10.1.2 Canada
    10.1.2.1 Market Trends
    10.1.2.2 Market Forecast
10.2 Asia-Pacific
  10.2.1 China
    10.2.1.1 Market Trends
    10.2.1.2 Market Forecast
  10.2.2 Japan
    10.2.2.1 Market Trends
    10.2.2.2 Market Forecast
  10.2.3 India
    10.2.3.1 Market Trends
    10.2.3.2 Market Forecast
  10.2.4 South Korea
    10.2.4.1 Market Trends
    10.2.4.2 Market Forecast
  10.2.5 Australia
    10.2.5.1 Market Trends
    10.2.5.2 Market Forecast
  10.2.6 Indonesia
    10.2.6.1 Market Trends
    10.2.6.2 Market Forecast
  10.2.7 Others
    10.2.7.1 Market Trends
    10.2.7.2 Market Forecast
10.3 Europe
  10.3.1 Germany
    10.3.1.1 Market Trends
    10.3.1.2 Market Forecast
  10.3.2 France
    10.3.2.1 Market Trends
    10.3.2.2 Market Forecast
  10.3.3 United Kingdom
    10.3.3.1 Market Trends
    10.3.3.2 Market Forecast
  10.3.4 Italy
    10.3.4.1 Market Trends
    10.3.4.2 Market Forecast
  10.3.5 Spain
    10.3.5.1 Market Trends
    10.3.5.2 Market Forecast
  10.3.6 Russia
    10.3.6.1 Market Trends
    10.3.6.2 Market Forecast
  10.3.7 Others
    10.3.7.1 Market Trends
    10.3.7.2 Market Forecast
10.4 Latin America
  10.4.1 Brazil
    10.4.1.1 Market Trends
    10.4.1.2 Market Forecast
  10.4.2 Mexico
    10.4.2.1 Market Trends
    10.4.2.2 Market Forecast
  10.4.3 Others
    10.4.3.1 Market Trends
    10.4.3.2 Market Forecast
10.5 Middle East and Africa
  10.5.1 Market Trends
  10.5.2 Market Breakup by Country
  10.5.3 Market Forecast

11 DRIVERS, RESTRAINTS, AND OPPORTUNITIES

11.1 Overview
11.2 Drivers
11.3 Restraints
11.4 Opportunities

12 VALUE CHAIN ANALYSIS


13 PORTERS FIVE FORCES ANALYSIS

13.1 Overview
13.2 Bargaining Power of Buyers
13.3 Bargaining Power of Suppliers
13.4 Degree of Competition
13.5 Threat of New Entrants
13.6 Threat of Substitutes

14 PRICE ANALYSIS


15 COMPETITIVE LANDSCAPE

15.1 Market Structure
15.2 Key Players
15.3 Profiles of Key Players
  15.3.1 Amicus Therapeutics Inc.
    15.3.1.1 Company Overview
    15.3.1.2 Product Portfolio
    15.3.1.3 Financials
    15.3.1.4 SWOT Analysis
  15.3.2 Audentes Therapeutics Inc. (Astellas US Holding Inc.)
    15.3.2.1 Company Overview
    15.3.2.2 Product Portfolio
  15.3.3 Oxyrane UK Limited
    15.3.3.1 Company Overview
    15.3.3.2 Product Portfolio
  15.3.4 Sanofi S.A.
    15.3.4.1 Company Overview
    15.3.4.2 Product Portfolio
    15.3.4.3 Financials
    15.3.4.4 SWOT Analysis
  15.3.5 Spark Therapeutics Inc.
    15.3.5.1 Company Overview
    15.3.5.2 Product Portfolio


LIST OF TABLES

Table 1: Global: Pompe Disease Treatment Market: Key Industry Highlights, 2023 & 2032
Table 2: Global: Pompe Disease Treatment Market Forecast: Breakup by Treatment (in Million US$), 2024-2032
Table 3: Global: Pompe Disease Treatment Market Forecast: Breakup by Route of Administration (in Million US$), 2024-2032
Table 4: Global: Pompe Disease Treatment Market Forecast: Breakup by Distribution Channel (in Million US$), 2024-2032
Table 5: Global: Pompe Disease Treatment Market Forecast: Breakup by Indication Type (in Million US$), 2024-2032
Table 6: Global: Pompe Disease Treatment Market Forecast: Breakup by Region (in Million US$), 2024-2032
Table 7: Global: Pompe Disease Treatment Market: Competitive Structure
Table 8: Global: Pompe Disease Treatment Market: Key Players

LIST OF FIGURES

Figure 1: Global: Pompe Disease Treatment Market: Major Drivers and Challenges
Figure 2: Global: Pompe Disease Treatment Market: Sales Value (in Billion US$), 2018-2023
Figure 3: Global: Pompe Disease Treatment Market Forecast: Sales Value (in Billion US$), 2024-2032
Figure 4: Global: Pompe Disease Treatment Market: Breakup by Treatment (in %), 2023
Figure 5: Global: Pompe Disease Treatment Market: Breakup by Route of Administration (in %), 2023
Figure 6: Global: Pompe Disease Treatment Market: Breakup by Distribution Channel (in %), 2023
Figure 7: Global: Pompe Disease Treatment Market: Breakup by Indication Type (in %), 2023
Figure 8: Global: Pompe Disease Treatment Market: Breakup by Region (in %), 2023
Figure 9: Global: Pompe Disease Treatment (Enzyme Replacement Therapy (ERT)) Market: Sales Value (in Million US$), 2018 & 2023
Figure 10: Global: Pompe Disease Treatment (Enzyme Replacement Therapy (ERT)) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 11: Global: Pompe Disease Treatment (Substrate Reduction Therapy (SRT)) Market: Sales Value (in Million US$), 2018 & 2023
Figure 12: Global: Pompe Disease Treatment (Substrate Reduction Therapy (SRT)) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 13: Global: Pompe Disease Treatment (Chaperone-Advanced Replacement Therapy (CART)) Market: Sales Value (in Million US$), 2018 & 2023
Figure 14: Global: Pompe Disease Treatment (Chaperone-Advanced Replacement Therapy (CART)) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 15: Global: Pompe Disease Treatment (Other Treatments) Market: Sales Value (in Million US$), 2018 & 2023
Figure 16: Global: Pompe Disease Treatment (Other Treatments) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 17: Global: Pompe Disease Treatment (Oral) Market: Sales Value (in Million US$), 2018 & 2023
Figure 18: Global: Pompe Disease Treatment (Oral) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 19: Global: Pompe Disease Treatment (Intravenous) Market: Sales Value (in Million US$), 2018 & 2023
Figure 20: Global: Pompe Disease Treatment (Intravenous) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 21: Global: Pompe Disease Treatment (Other Rout of Administrations) Market: Sales Value (in Million US$), 2018 & 2023
Figure 22: Global: Pompe Disease Treatment (Other Rout of Administrations) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 23: Global: Pompe Disease Treatment (Hospital & Clinics Pharmacies) Market: Sales Value (in Million US$), 2018 & 2023
Figure 24: Global: Pompe Disease Treatment (Hospital & Clinics Pharmacies) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 25: Global: Pompe Disease Treatment (Retail Pharmacies) Market: Sales Value (in Million US$), 2018 & 2023
Figure 26: Global: Pompe Disease Treatment (Retail Pharmacies) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 27: Global: Pompe Disease Treatment (Online Pharmacies) Market: Sales Value (in Million US$), 2018 & 2023
Figure 28: Global: Pompe Disease Treatment (Online Pharmacies) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 29: Global: Pompe Disease Treatment (Other Distribution Channels) Market: Sales Value (in Million US$), 2018 & 2023
Figure 30: Global: Pompe Disease Treatment (Other Distribution Channels) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 31: Global: Pompe Disease Treatment (Infantile-Onset Pompe Disease (IOPD)) Market: Sales Value (in Million US$), 2018 & 2023
Figure 32: Global: Pompe Disease Treatment (Infantile-Onset Pompe Disease (IOPD)) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 33: Global: Pompe Disease Treatment (Classic Infantile Form) Market: Sales Value (in Million US$), 2018 & 2023
Figure 34: Global: Pompe Disease Treatment (Classic Infantile Form) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 35: Global: Pompe Disease Treatment (Non-Classic Infantile Form) Market: Sales Value (in Million US$), 2018 & 2023
Figure 36: Global: Pompe Disease Treatment (Non-Classic Infantile Form) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 37: Global: Pompe Disease Treatment (Late-Onset Pompe Disease (LOPD)) Market: Sales Value (in Million US$), 2018 & 2023
Figure 38: Global: Pompe Disease Treatment (Late-Onset Pompe Disease (LOPD)) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 39: Global: Pompe Disease Treatment (Other Indication Types) Market: Sales Value (in Million US$), 2018 & 2023
Figure 40: Global: Pompe Disease Treatment (Other Indication Types) Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 41: North America: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 42: North America: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 43: United States: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 44: United States: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 45: Canada: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 46: Canada: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 47: Asia-Pacific: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 48: Asia-Pacific: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 49: China: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 50: China: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 51: Japan: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 52: Japan: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 53: India: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 54: India: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 55: South Korea: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 56: South Korea: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 57: Australia: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 58: Australia: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 59: Indonesia: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 60: Indonesia: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 61: Others: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 62: Others: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 63: Europe: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 64: Europe: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 65: Germany: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 66: Germany: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 67: France: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 68: France: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 69: United Kingdom: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 70: United Kingdom: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 71: Italy: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 72: Italy: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 73: Spain: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 74: Spain: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 75: Russia: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 76: Russia: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 77: Others: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 78: Others: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 79: Latin America: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 80: Latin America: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 81: Brazil: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 82: Brazil: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 83: Mexico: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 84: Mexico: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 85: Others: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 86: Others: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 87: Middle East and Africa: Pompe Disease Treatment Market: Sales Value (in Million US$), 2018 & 2023
Figure 88: Middle East and Africa: Pompe Disease Treatment Market: Breakup by Country (in %), 2023
Figure 89: Middle East and Africa: Pompe Disease Treatment Market Forecast: Sales Value (in Million US$), 2024-2032
Figure 90: Global: Pompe Disease Treatment Industry: Drivers, Restraints, and Opportunities
Figure 91: Global: Pompe Disease Treatment Industry: Value Chain Analysis
Figure 92: Global: Pompe Disease Treatment Industry: Porter's Five Forces Analysis


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