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ポンペ病治療市場：治療別、投与経路別、流通チャネル別、適応症タイプ別、地域別、2024-2032年

Pompe Disease Treatment Market by Treatment, Route of Administration, Distribution Channel, Indication Type, and Region 2024-2032
出版日: | 発行: IMARC | ページ情報: 英文 142 Pages | 納期: 2～3営業日

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価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=157.53円
ポンペ病治療市場：治療別、投与経路別、流通チャネル別、適応症タイプ別、地域別、2024-2032年
出版日: 2024年01月30日
発行: IMARC
ページ情報: 英文 142 Pages
納期: 2～3営業日
  • 全表示
  • 概要
  • 図表
  • 目次
概要

世界のポンペ病治療市場規模は2023年に10億米ドルに達しました。今後、IMARC Groupは、2024年から2032年にかけて2.58%の成長率（CAGR）を示し、2032年までに13億米ドルに達すると予測しています。臨床研究および臨床試験の増加、ERTの人気上昇、ポンペ病に罹患する患者数の増加が市場を牽引する主な要因のいくつかです。

ポンペ病は、体内の細胞内にグリコーゲンとして知られる複合糖質が蓄積することによって引き起こされる遺伝病であり、臓器、組織、筋肉に影響を及ぼします。常染色体劣性遺伝のグリコーゲン蓄積症候群で、ライソゾーム酵素である酸性a-グルコシダーゼ（GAA）の欠損によって引き起こされます。筋力低下、肝臓肥大、呼吸困難、聴力障害、肺感染、不整脈、体重減少などを伴います。ポンペ病は、筋電図、X線、磁気共鳴画像法（MRI）など、さまざまな手段や検査によって診断されます。ポンペ病は、薬物療法、酵素補充療法（ERT）、基質減少療法（SRT）、シャペロン高度補充療法（CART）、注射療法などを用いて治療することができ、生活の質を改善するのに役立ちます。

ポンペ病治療市場の動向：

個人における遺伝子異常の有病率が大幅に上昇し、ポンペ病に罹患する患者数が増加しています。このことは、乳幼児の間でポンペ病が増加し、新生児の健康に対する両親の関心が高まっていることと相まって、市場の成長を後押しする重要な要因の1つとなっています。さらに、これらの医学的障害を発症しやすい老年人口の増加が、ポンペ病治療に対する需要の増加をもたらしています。さらに、ポンペ病の新しい治療法を開発するための臨床研究や臨床試験の数が拡大していることも、市場にプラスの影響を与えています。これとは別に、ポンペ病を治すためのERTに関する人々の意識の高まりが、市場の成長に好影響を与えています。さらに、幹細胞移植、骨髄移植、化学療法、免疫療法など、さまざまな治療法に対する認知度が高まっています。これに加えて、ルミザイムなどのERT治療代替薬が容易に入手可能であることも認知され、市場には明るい展望が生まれています。これに加えて、ポンペ病治療のための経済的支援を提供し、医療支出を最小限に抑える健康保険政策の採用が増加しています。さらに、各国の政府や非政府組織（NGO）は、誰もが利用できる質の高い医療施設を提供するための対策に取り組んでいます。このことは、診断技術の大幅な改善とともに、市場に良好な見通しをもたらすと予想されます。

本レポートで扱う主な質問

  • 世界のポンペ病治療市場はこれまでどのように推移し、今後どのように推移していくのか？
  • 世界のポンペ病治療市場における市場促進要因、市場抑制要因、機会は何か？
  • 主要な地域市場は？
  • 最も魅力的なポンペ病治療市場はどの国か？
  • 治療に基づく市場内訳は？
  • 投与経路別の市場内訳は？
  • 販売チャネル別の市場内訳は？
  • 適応症タイプ別の市場内訳は？
  • 世界のポンペ病治療市場の競争構造は？
  • 世界のポンペ病治療薬市場における主要プレイヤー/企業は？

目次

第1章 序文

第2章 調査範囲と調査手法

  • 調査目的
  • 利害関係者
  • データソース
    • 一次情報
    • 二次情報
  • 市場推定
    • ボトムアップアプローチ
    • トップダウンアプローチ
  • 調査手法

第3章 エグゼクティブサマリー

第4章 イントロダクション

  • 概要
  • 主要産業動向

第5章 ポンペ病治療の世界市場

  • 市場概要
  • 市場実績
  • COVID-19の影響
  • 市場予測

第6章 市場内訳：治療別

  • 酵素補充療法（ERT）
    • 市場動向
    • 市場予測
  • 基質低減療法（SRT）
    • 市場動向
    • 市場予測
  • シャペロン先進補充療法（CART）
    • 市場動向
    • 市場予測
  • その他
    • 市場動向
    • 市場予測

第7章 市場内訳：投与経路別

  • 経口
    • 市場動向
    • 市場予測
  • 静脈内
    • 市場動向
    • 市場予測
  • その他
    • 市場動向
    • 市場予測

第8章 市場内訳：流通チャネル別

  • 病院・クリニック薬局
    • 市場動向
    • 市場予測
  • 小売薬局
    • 市場動向
    • 市場予測
  • オンライン薬局
    • 市場動向
    • 市場予測
  • その他
    • 市場動向
    • 市場予測

第9章 市場内訳：適応症タイプ別

  • 小児発症ポンぺ病（IOPD）
    • 市場動向
    • 市場予測
  • 古典的乳児型ポンペ病
    • 市場動向
    • 市場予測
  • 非古典的乳児型ポンペ病
    • 市場動向
    • 市場予測
  • 遅発性ポンペ病（LOPD）
    • 市場動向
    • 市場予測
  • その他
    • 市場動向
    • 市場予測

第10章 市場内訳：地域別

  • 北米
    • 米国
      • 市場動向
      • 市場予測
    • カナダ
      • 市場動向
      • 市場予測
  • アジア太平洋
    • 中国
      • 市場動向
      • 市場予測
    • 日本
      • 市場動向
      • 市場予測
    • インド
      • 市場動向
      • 市場予測
    • 韓国
      • 市場動向
      • 市場予測
    • オーストラリア
      • 市場動向
      • 市場予測
    • インドネシア
      • 市場動向
      • 市場予測
    • その他
      • 市場動向
      • 市場予測
  • 欧州
    • ドイツ
      • 市場動向
      • 市場予測
    • フランス
      • 市場動向
      • 市場予測
    • 英国
      • 市場動向
      • 市場予測
    • イタリア
      • 市場動向
      • 市場予測
    • スペイン
      • 市場動向
      • 市場予測
    • ロシア
      • 市場動向
      • 市場予測
    • その他
      • 市場動向
      • 市場予測
  • ラテンアメリカ
    • ブラジル
      • 市場動向
      • 市場予測
    • メキシコ
      • 市場動向
      • 市場予測
    • その他
      • 市場動向
      • 市場予測
  • 中東・アフリカ地域
    • 市場動向
    • 市場内訳：国別
    • 市場予測

第11章 促進要因、抑制要因、機会

  • 市場概要
  • 促進要因
  • 抑制要因
  • 機会

第12章 バリューチェーン分析

第13章 ポーターのファイブフォース分析

  • 概要
  • 買い手の交渉力
  • 供給企業の交渉力
  • 競合の程度
  • 新規参入業者の脅威
  • 代替品の脅威

第14章 価格分析

第15章 競合情勢

  • 市場構造
  • 主要企業
  • 主要企業のプロファイル
    • Amicus Therapeutics Inc.
    • Audentes Therapeutics Inc.（Astellas US Holding Inc.）
    • Oxyrane UK Limited
    • Sanofi S.A.
    • Spark Therapeutics Inc.
図表

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

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
目次
Product Code: SR112024A6708

Abstract

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?

Table of Contents

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

Kindly, note that this only represents a partial list of companies, and the complete list has been provided in the report