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폼페병(PD) : 역학 예측(-2030년)

Pompe Disease - Epidemiology Forecast to 2030

리서치사 DelveInsight Business Research LLP
발행일 2020년 05월 상품 코드 938690
페이지 정보 영문 100 Pages
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US $ 3,750 ₩ 4,314,000 PDF by E-mail (Single User License)
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폼페병(PD) : 역학 예측(-2030년) Pompe Disease - Epidemiology Forecast to 2030
발행일 : 2020년 05월 페이지 정보 : 영문 100 Pages

본 상품은 영문 자료로 한글과 영문목차에 불일치하는 내용이 있을 경우 영문을 우선합니다. 정확한 검토를 위해 영문목차를 참고해주시기 바랍니다.

주요 7개국(미국, 일본, 독일, 스페인, 이탈리아, 프랑스, 영국)의 폼페병(PD: Pompe Disease) 관련 시장을 조사했으며, 시장 개요, 역학과 환자수, 국가별 역학, 치료 및 관리, 사례 연구, 충족되지 않은 욕구 등의 정보를 제공합니다.

목차

제1장 주요 조사결과

제2장 질환 개요

제3장 SWOT 분석

제4장 질환의 배경과 개요

  • 서론
  • 유형
  • 유전적 결함
  • 임상 증상
  • 원인
  • 징후 및 증상
  • 발병기전
  • 진단

제5장 역학과 환자수

  • 주요 조사결과
  • 7개국의 총 환자수

제6장 폼페병의 국가별 역학

  • 미국
    • 전제조건과 근거
    • 신생아 환자수
    • 성인 환자수
    • 총 환자수
    • 발증 유형별 환자수
    • 임상 표현형별 환자수
  • 유럽 5개국
    • 독일
    • 프랑스
    • 이탈리아
    • 스페인
    • 영국
  • 일본

제7장 치료 및 관리

  • 효소대체요법(ERT)

제8장 유전자 치료 : 향후 전망

  • AAV 벡터
  • 유전자 치료와 효소대체요법(ERT)
  • 유전자 치료의 과제 : 생산과 면역반응
    • 임상 응용을 위한 Recombinant Adeno-Associated Vector 제작
    • AAV Capsid and the Transgene에 대한 면역반응과 벡터 재투여

제9장 사례 연구

제10장 환자 여정

제11장 충족되지 않은 욕구

제12장 부록

  • 보고서 조사 방법

제13장 DelveInsight의 서비스 내용

제14장 면책조항

제15장 DelveInsight에 대해

LSH 20.06.19

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

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

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