市場調查報告書

龐貝氏症(PD):主要7個國家(美國,日本,德國,西班牙,義大利,法國,英國)到2030年前的市場分析,流行病學,市場預測

Pompe Disease - Epidemiology Forecast to 2030

出版商 DelveInsight Business Research LLP 商品編碼 938690
出版日期 內容資訊 英文 100 Pages
商品交期: 最快1-2個工作天內
價格
龐貝氏症(PD):主要7個國家(美國,日本,德國,西班牙,義大利,法國,英國)到2030年前的市場分析,流行病學,市場預測 Pompe Disease - Epidemiology Forecast to 2030
出版日期: 2020年05月01日內容資訊: 英文 100 Pages
簡介

本報告提供主要7個國家(美國,日本,德國,西班牙,義大利,法國,英國)的龐貝氏症(PD)相關市場調查,提供市場概要,流行病學患者數,各國流行病學,治療和管理,案例研究,未支援的需求等資訊。

目錄

第1章 主要調查結果

第2章 疾病概要

第3章 SWOT分析

第4章 疾病的背景和概要

  • 簡介
  • 類型
  • 遺傳的缺點
  • 臨床症狀
  • 原因
  • 徵兆與症狀
  • 發病機制
  • 診斷

第5章 流行病學和患者數

  • 主要調查結果
  • 7個國家的整體患病人數

第6章 龐貝氏症的各國流行病學

  • 美國
    • 前提和根據
    • 新生兒的患病人數
    • 成人的患病人數
    • 整體患病人數
    • 各發病型的患病人數
    • 各臨床表現型的患病人數
  • 歐洲5個國家
    • 德國
    • 法國
    • 義大利
    • 西班牙
    • 英國
  • 日本

第7章 治療和管理

  • 酵素補充療法(ERT)

第8章 基因治療:未來展望

  • AAV媒介
  • 基因治療和ERT
  • 基因治療的課題:生產與免疫反應
    • 用於臨床重組腺相關載體的生產
    • 對AAV衣殼和轉基因的免疫反應,以及載體的重新管理

第9章 案例研究

第10章 病人旅程

第11章 未支援的需求

第12章 附錄

  • 報告調查手法

第13章 DelveInsight的服務內容

第14章 免責聲明

第15章 關於DelveInsight

目錄
Product Code: DIEI0177

DelveInsight's 'Pompe Disease (PD) - Epidemiology Forecast to 2030' report delivers an in-depth understanding of the disease, historical & forecasted epidemiology of Pompe Disease in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom) and Japan.

Pompe Disease (PD) Understanding

Pompe disease (PD), also known as glycogen storage disease type II (GSDII) or "acid maltase deficiency", is caused by the absence or deficiency of acid alpha-glucosidase (GAA), a lysosomal enzyme that is responsible for the cleavage of the α-1, 4- and α-1, 6-glycosidic bonds of glycogen to glucose. The disease is caused pathogenic variations in the acid alpha-glucosidase (GAA) gene. Close to 500 different GAA gene variations have been identified in families with this disorder.

Pompe Disease Epidemiology Perspective by DelveInsight

The Pompe Disease (PD) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every seven major countries. The epidemiology data for Pompe Disease are studied through all possible division to give a better understanding about the Disease scenario in 7MM. It also helps to recognize the causes of current and forecasted trends by exploring numerous studies, survey reports and views of key opinion leaders.

Pompe Disease Detailed Epidemiology Segmentation

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes) scenario of Pompe Disease (PD) in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017-2030.

The DelveInsight PD report also provides the epidemiology trends observed in the 7MM during the study period, along with the assumptions undertaken. The calculated data are presented with relevant tables and graphs to give a clear view of the epidemiology at first sight.

Scope of the Report:

  • The report covers detailed overview of Pompe Disease explaining its causes, symptoms, classification, pathophysiology, diagnosis and treatment patterns
  • The Pompe Disease (PD) Report and Model provide an overview of the risk factors and global trends of PD in the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan)
  • The report provides the insight about the historical and forecasted patient pool of Pompe Disease in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) and Japan
  • The Report assesses the disease risk and burden and highlights the unmet needs of the disease
  • The Report helps to recognize the growth opportunities in the 7MM with respect to the patient population
  • The report provides the segmentation of the disease epidemiology by Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes in 7MM

Report Highlights:

  • 11 Years Forecast
  • 7MM Coverage
  • Total Diagnosed Prevalent Cases of Pompe Disease
  • Prevalent Cases according to segmentation: Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes

KOL- Views

We interview, KOLs and SME's opinion through primary research to fill the data gaps and validate our secondary research. The opinion helps to understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.

Key Questions Answered

  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Pompe Disease?
  • What are the key findings pertaining to the Pompe Disease epidemiology across 7MM and which country will have the highest number of patients during the forecast period (2020-2030)?
  • What would be the total number of patients of Pompe Disease across the 7MM during the forecast period (2020-2030)?
  • Among the EU5 countries, which country will have the highest number of patients during the forecast period (2020-2030)?
  • At what CAGR the patient population is expected to grow in 7MM during the forecast period (2020-2030)?
  • What is the disease risk, burden and unmet needs of the Pompe Disease?
  • What are the current available treatments of Pompe Disease?

Reasons to buy:

The Pompe Disease Epidemiology report will allow the user to -

  • Develop business strategies by understanding the trends shaping and driving the global Pompe Disease market
  • Quantify patient populations in the global Pompe Disease market to improve product design, pricing, and launch plans
  • Organize sales and marketing efforts by identifying the age groups that present the best opportunities for Pompe Disease therapeutics in each of the markets covered
  • Understand the magnitude of Pompe Disease population by its severity
  • The Pompe Disease epidemiology report and model were written and developed by Masters and PhD level epidemiologists
  • The Pompe Disease Epidemiology Model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based with transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over 11-year forecast period using reputable sources

Key Assessments

  • Patient Segmentation
  • Disease Risk and Burden
  • Risk of disease by the segmentation
  • Factors driving growth in a specific patient population

Geographies Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2017-2030

Although Pompe disease is a rare autosomal recessive disorder there have been attempts at estimating the overall worldwide incidence with results yielding a value of ~1/40,000. Organizations such as the National Organization for Rare Disorders (NORD) and United Pompe Foundation have also quoted such figures in their publications and journals. However, a fear of underestimation has always prevailed despite adequate precautionary measures due to the inherent difficulty in diagnosis

Newborn screening studies have forced experts to revise previously accepted incidence/prevalence numbers and DelveInsight has kept its focus on these recent happenings while formulating the Epidemiology model for 7 MM

Table of Contents

1. Key Insights

2. Executive Summary of Pompe Disease (PD)

3. SWOT Analysis for Pompe Disease

4. Disease Background and Overview

  • 4.1. Introduction
  • 4.2. Types
  • 4.3. Genetic defects
  • 4.4. Clinical Manifestations
  • 4.5. Causes
  • 4.6. Signs and Symptoms
  • 4.7. Pathogenesis
  • 4.8. Diagnosis

5. Epidemiology and Patient Population

  • 5.1. Key Findings
  • 5.2. 7MM Total Prevalent Patient Population of Pompe Disease

6. Country Wise-Epidemiology of Pompe Disease

  • 6.1. The United States
    • 6.1.1. Assumptions and Rationale
    • 6.1.2. Total Live Birth Cases of Pompe Disease in the US
    • 6.1.3. Total Adult Prevalent Cases of Pompe Disease in the US
    • 6.1.4. Total Prevalent Cases of Pompe Disease in the US
    • 6.1.5. Prevalence of Pompe Disease by Onset Types in the US
    • 6.1.6. Incidence of IOPD based on Clinical Phenotype in the US
  • 6.2. EU5
    • 6.2.1. Assumptions and Rationale
  • 6.3. Germany
    • 6.3.1. Total Live Birth Cases of Pompe Disease in Germany
    • 6.3.2. Total Adult Prevalent Cases of Pompe Disease in Germany
    • 6.3.3. Total Prevalent Cases of Pompe Disease in Germany
    • 6.3.4. Prevalence of Pompe Disease by Onset Types in Germany
    • 6.3.5. Incidence of IOPD based on Clinical Phenotype in Germany
  • 6.4. France
    • 6.4.1. Total Live Birth Cases of Pompe Disease in France
    • 6.4.2. Total Adult Prevalent Cases of Pompe Disease in France
    • 6.4.3. Total Prevalent Cases of Pompe Disease in France
    • 6.4.4. Prevalence of Pompe Disease by Onset Types in France
    • 6.4.5. Incidence of IOPD based on Clinical Phenotype in France
  • 6.5. Italy
    • 6.5.1. Total Live Birth Cases of Pompe Disease in Italy
    • 6.5.2. Total Adult Prevalent Cases of Pompe Disease in Italy
    • 6.5.3. Total Prevalent Cases of Pompe Disease in Italy
    • 6.5.4. Prevalence of Pompe Disease by Onset Types in Italy
    • 6.5.5. Incidence of IOPD based on Clinical Phenotype in Italy
  • 6.6. Spain
    • 6.6.1. Total Live Birth Cases of Pompe Disease in Spain
    • 6.6.2. Total Adult Prevalent Cases of Pompe Disease in Spain
    • 6.6.3. Total Prevalent Cases of Pompe Disease in Spain
    • 6.6.4. Prevalence of Pompe Disease by Onset Types in Spain
    • 6.6.5. Incidence of IOPD based on Clinical Phenotype in Spain
  • 6.7. The United Kingdom
    • 6.7.1. Total Live Birth Cases of Pompe Disease in the UK
    • 6.7.2. Total Adult Prevalent Cases of Pompe Disease in the UK
    • 6.7.3. Total Prevalent Cases of Pompe Disease in the UK
    • 6.7.4. Prevalence of Pompe Disease by Onset Types in the UK
    • 6.7.5. Incidence of IOPD based on Clinical Phenotype in the UK
  • 6.8. Japan
    • 6.8.1. Assumptions and Rationale
    • 6.8.2. Total Live Birth Cases of Pompe Disease in Japan
    • 6.8.3. Total Adult Prevalent Cases of Pompe Disease in Japan
    • 6.8.4. Total Prevalent Cases of Pompe Disease in Japan
    • 6.8.5. Prevalence of Pompe Disease by Onset Types in Japan
    • 6.8.6. Incidence of IOPD based on Clinical Phenotype in Japan

7. Treatment and Management of Pompe Disease

  • 7.1. Enzyme Replacement Therapy in Pompe Disease

8. Gene Therapy: What Future Holds

  • 8.1. AAV Vectors
  • 8.2. Gene therapy vs ERT
  • 8.3. Challenges in Gene Therapy: Production & Immune Response
    • 8.3.1. Production of Recombinant Adeno-Associated Vectors for Clinical Application
    • 8.3.2. Immune-Responses to AAV Capsid and the Transgene, and Vector Re-Administration

9. Case Study

  • 9.1. Pompe Disease: Presentation and Management of Early Onset Type with Perioperative Considerations

10. Patient Journey

11. Unmet Needs

12. Appendix

  • 12.1. Report Methodology

13. DelveInsight Capabilities

14. Disclaimer

15. About DelveInsight

List of Tables

  • Table 1: Summary of Pompe Disease (PD) Market, Epidemiology, and Key Events (2017-2030)
  • Table 2: Prevalent Patient Population of Pompe Disease in 7MM (2017-2030)
  • Table 3: Total Live Birth Cases of Pompe Disease in the US (2017-2030)
  • Table 4: Adult Prevalent Cases of Pompe Disease in the United States (2017-2030)
  • Table 5: Total Diagnosed Prevalent Cases of Pompe Disease in the United States (2017-2030)
  • Table 6: Diagnosed Prevalence of Pompe Disease by Onset Types in the United States (2017-2030)
  • Table 7 Incidence of IOPD based on Clinical Phenotypes in the United States (2017-2030)
  • Table 8: Total Live Birth Cases of Pompe Disease in Germany (2017-2030)
  • Table 9: Adult Prevalent Cases of Pompe Disease in Germany (2017-2030)
  • Table 10: Total Diagnosed Prevalent Cases of Pompe Disease in Germany (2017-2030)
  • Table 11: Diagnosed Prevalence of Pompe Disease by Onset Types in Germany (2017-2030)
  • Table 12 Incidence of IOPD based on Clinical Phenotypes in Germany (2017-2030)
  • Table 13: Total Live Birth Cases of Pompe Disease in France (2017-2030)
  • Table 14: Adult Prevalent Cases of Pompe Disease in France (2017-2030)
  • Table 15: Total Diagnosed Prevalent Cases of Pompe Disease in France (2017-2030)
  • Table 16: Diagnosed Prevalence of Pompe Disease by Onset Types in France (2017-2030)
  • Table 17 Incidence of IOPD based on Clinical Phenotypes in France (2017-2030)
  • Table 18: Total Live Birth Cases of Pompe Disease in Italy (2017-2030)
  • Table 19: Adult Prevalent Cases of Pompe Disease in Italy (2017-2030)
  • Table 20: Total Diagnosed Prevalent Cases of Pompe Disease in Italy (2017-2030)
  • Table 21: Diagnosed Prevalence of Pompe Disease by Onset Types in Italy (2017-2030)
  • Table 22 Incidence of IOPD based on Clinical Phenotypes in Italy (2017-2030)
  • Table 23: Total Live Birth Cases of Pompe Disease in Spain (2017-2030)
  • Table 24: Adult Prevalent Cases of Pompe Disease in Spain (2017-2030)
  • Table 25: Total Diagnosed Prevalent Cases of Pompe Disease in Spain (2017-2030)
  • Table 26: Diagnosed Prevalence of Pompe Disease by Onset Types in Spain (2017-2030)
  • Table 27 Incidence of IOPD based on Clinical Phenotypes in Spain (2017-2030)
  • Table 28: Total Live Birth Cases of Pompe Disease in the UK (2017-2030)
  • Table 29: Adult Prevalent Cases of Pompe Disease in the UK (2017-2030)
  • Table 30: Total Diagnosed Prevalent Cases of Pompe Disease in the UK (2017-2030)
  • Table 31: Diagnosed Prevalence of Pompe Disease by Onset Types in the UK (2017-2030)
  • Table 32 Incidence of IOPD based on Clinical Phenotypes in the UK (2017-2030)
  • Table 33: Total Live Birth Cases of Pompe Disease in Japan (2017-2030)
  • Table 34: Adult Prevalent Cases of Pompe Disease in Japan (2017-2030)
  • Table 35: Total Diagnosed Prevalent Cases of Pompe Disease in Japan (2017-2030)
  • Table 36: Diagnosed Prevalence of Pompe Disease by Onset Types in Japan (2017-2030)
  • Table 37 Incidence of IOPD based on Clinical Phenotypes in Japan (2017-2030)
  • Table 38: AT-GAA, Clinical Trial Description, 2020
  • Table 39: AT-GAA, Clinical Trial Description, 2020
  • Table 40: ACTUS-101, Clinical Trial Description, 2020
  • Table 41: VAL-1221, Clinical Trial Description, 2020
  • Table 42:AAV9, Clinical Trial Description, 2020
  • Table 43: ACTUS-101, Clinical Trial Description, 2020
  • Table 44:AAV9, Clinical Trial Description, 2020

List of Figures

  • Figure 1: SWOT Analysis
  • Figure 2: Comparison of normal physiology to pathophysiology in Pompe disease
  • Figure 3: Pathophysiology of late-onset Pompe disease
  • Figure 4: Diagnostic algorithm for infantile-onset Pompe disease.
  • Figure 5: Diagnostic algorithm for late-onset (> 1 year) Pompe disease.
  • Figure 6: Prevalent Patient Population of Pompe Disease in 7MM (2017-2030)
  • Figure 7: Total Live Birth Cases of Pompe Disease in the US (2017-2030)
  • Figure 8: Adult Prevalent Cases of Pompe Disease in the United States (2017-2030)
  • Figure 9: Total Diagnosed Prevalent Cases of Pompe Disease in the United States (2017-2030)
  • Figure 10: Diagnosed Prevalence of Pompe Disease by Onset Types in the United States (2017-2030)
  • Figure 11: Incidence of IOPD based on Clinical Phenotypes in the United States (2017-2030)
  • Figure 12: Total Live Birth Cases of Pompe Disease in Germany (2017-2030)
  • Figure 13: Total Diagnosed Prevalent Cases of Pompe Disease in Germany (2017-2030)
  • Figure 14: Total Diagnosed Prevalent Cases of Pompe Disease in Germany (2017-2030)
  • Figure 15: Diagnosed Prevalence of Pompe Disease by Onset Types in Germany (2017-2030)
  • Figure 16: Incidence of IOPD based on Clinical Phenotypes in Germany (2017-2030)
  • Figure 17: Total Live Birth Cases of Pompe Disease in France (2017-2030)
  • Figure 18: Total Diagnosed Prevalent Cases of Pompe Disease in France (2017-2030)
  • Figure 19: Total Diagnosed Prevalent Cases of Pompe Disease in France (2017-2030)
  • Figure 20: Diagnosed Prevalence of Pompe Disease by Onset Types in France (2017-2030)
  • Figure 21: Incidence of IOPD based on Clinical Phenotypes in France (2017-2030)
  • Figure 22: Total Live Birth Cases of Pompe Disease in Italy (2017-2030)
  • Figure 23: Total Diagnosed Prevalent Cases of Pompe Disease in Italy (2017-2030)
  • Figure 24: Total Diagnosed Prevalent Cases of Pompe Disease in Italy (2017-2030)
  • Figure 25: Diagnosed Prevalence of Pompe Disease by Onset Types in Italy (2017-2030)
  • Figure 26: Incidence of IOPD based on Clinical Phenotypes in Italy (2017-2030)
  • Figure 27: Total Live Birth Cases of Pompe Disease in Spain (2017-2030)
  • Figure 28: Total Diagnosed Prevalent Cases of Pompe Disease in Spain (2017-2030)
  • Figure 29: Total Diagnosed Prevalent Cases of Pompe Disease in Spain (2017-2030)
  • Figure 30: Diagnosed Prevalence of Pompe Disease by Onset Types in Spain (2017-2030)
  • Figure 31: Incidence of IOPD based on Clinical Phenotypes in Spain (2017-2030)
  • Figure 32: Total Live Birth Cases of Pompe Disease in the UK (2017-2030)
  • Figure 33: Total Diagnosed Prevalent Cases of Pompe Disease in the UK (2017-2030)
  • Figure 34: Total Diagnosed Prevalent Cases of Pompe Disease in the UK (2017-2030)
  • Figure 35: Diagnosed Prevalence of Pompe Disease by Onset Types in the UK (2017-2030)
  • Figure 36: Incidence of IOPD based on Clinical Phenotypes in the UK (2017-2030)
  • Figure 37: Total Live Birth Cases of Pompe Disease in Japan (2017-2030)
  • Figure 38: Total Diagnosed Prevalent Cases of Pompe Disease in Japan (2017-2030)
  • Figure 39: Total Diagnosed Prevalent Cases of Pompe Disease in Japan (2017-2030)
  • Figure 40: Diagnosed Prevalence of Pompe Disease by Onset Types in Japan (2017-2030)
  • Figure 41: Incidence of IOPD based on Clinical Phenotypes in Japan (2017-2030)
  • Figure 42: Patient journey Pompe Disease
  • Figure 43: Unmet Needs of Pompe Disease