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アラジール症候群:疫学予測 (~2030年)

Alagille syndrome - Epidemiology Forecast-2030

出版日: | 発行: DelveInsight Business Research LLP | ページ情報: 英文 80 Pages | 納期: 即日から翌営業日

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アラジール症候群:疫学予測 (~2030年)
出版日: 2020年08月01日
発行: DelveInsight Business Research LLP
ページ情報: 英文 80 Pages
納期: 即日から翌営業日
担当者のコメント
遺伝性肝内胆汁うっ滞症の先天性疾患「アラジール症候群」の今後10年における疫学動向に着目した調査レポートが、Delveinsight社よりリリースされました。サンプルページをまずは送付させていただきますので、お問い合わせください。
  • 全表示
  • 概要
  • 図表
  • 目次
概要

アラジール症候群はJAG1またはNOTCH 2遺伝子の変異によって引き起こされる遺伝性疾患であり、胆汁うっ滞、黄疸、そう痒、先天性心疾患、腎疾患、骨疾患などの症状を伴い、合併症のため、多くの患者が肝移植を必要とします。3万人に1人の発生率のまれな疾患であり、表現型の変動性が高いことが特徴です。信頼できるバイオマーカーが入手できないため、診断が困難であり、また、利用可能な現在の治療法は対症療法であることから、病気の根本的な原因に対処していません。

当レポートでは、米国、欧州5カ国 (ドイツ・スペイン・イタリア・フランス・英国)、日本におけるアラジール症候群の疫学的動向を調査し、疾患概要、原因、症状、分類、病態生理、診断、治療パターン、有病者数の推移・予測、国・性別・年齢層別の内訳、KOLの見解などをまとめています。

第1章 主な洞察

第2章 エグゼクティブサマリー

第3章 組織

第4章 疫学的調査・調査手法

第5章 アラジール症候群:市場概要

  • シェア分布:実績
  • シェア分布:予測

第6章 アラジール症候群:市場概要

  • イントロダクション
  • 原因
  • 兆候・症状
  • 臨床所見
  • 病態生理
  • 予後
  • 診断

第7章 疫学・患者人口

  • 主な調査結果
  • 主要7カ国の全症例数
  • 米国
    • 有病者数
    • 有病者数:男女別
    • 有病者数:年齢層別
  • 欧州5カ国
  • ドイツ
    • 有病者数
    • 有病者数:男女別
    • 有病者数:年齢層別
  • フランス
    • 有病者数:年齢層別
  • イタリア
  • スペイン
    • 有病者数:年齢層別
  • 英国
    • 有病者数
    • 有病者数:男女別
    • 有病者数:年齢層別
  • 日本

第8章 KOLの見解

第9章 付録

第10章 調査手法

第11章 DelveInsightのサービス内容

第12章 免責事項

第13章 DelveInsightについて

図表

List of Tables

  • Table 1: Features of Alagille syndrome
  • Table 2: Molecular genetic testing used in Alagille syndrome
  • Table 3: Recommended evaluations following initial diagnosis in individuals with Alagille syndrome
  • Table 4: Classic criteria, based on five body systems, for a diagnosis of Alagille syndrome
  • Table 5: Genes of interest in the differential diagnosis of Alagille syndrome (ALGS)
  • Table 6: Total cases of Alagille syndrome in 7MM (2017-2030)
  • Table 7: Total prevalent cases of Alagille syndrome in the United States (2017-2030)
  • Table 8: Gender-specific cases of Alagille syndrome in the US (2017-2030)
  • Table 9: Age-specific cases of Alagille syndrome in the US (2017-2030)
  • Table 10: Total prevalent cases of Alagille syndrome in Germany (2017-2030)
  • Table 11: Gender-specific cases of Alagille syndrome in Germany (2017-2030)
  • Table 12: Age-specific cases of Alagille syndrome in Germany (2017-2030)
  • Table 13: Total prevalent cases of Alagille syndrome in France (2017-2030)
  • Table 14: Gender-specific cases of Alagille syndrome in France (2017-2030)
  • Table 15: Age-specific cases of Alagille syndrome in France (2017-2030)
  • Table 16: Total prevalent cases of Alagille syndrome in Italy (2017-2030)
  • Table 17: Gender-specific cases of Alagille syndrome in Italy (2017-2030)
  • Table 18: Age-specific cases of Alagille syndrome in Italy (2017-2030)
  • Table 19: Total prevalent cases of Alagille syndrome in Spain (2017-2030)
  • Table 20: Gender-specific cases of Alagille syndrome in Spain (2017-2030)
  • Table 21: Age-specific cases of Alagille syndrome in Spain (2017-2030)
  • Table 22: Total prevalent cases of Alagille syndrome in the United Kingdom (2017-2030)
  • Table 23: Gender-specific cases of Alagille syndrome in the UK (2017-2030)
  • Table 24: Age-specific cases of Alagille syndrome in the UK (2017-2030)
  • Table 25: Total prevalent cases of Alagille syndrome in Japan (2017-2030)
  • Table 26: Gender-specific cases of Alagille syndrome in Japan (2017-2030)
  • Table 27: Age-specific cases of Alagille syndrome in Japan (2017-2030)

List of Figures

  • Figure 1: Epidemiology and market methodology
  • Figure 2: Flow diagram of genetic investigations and management for suspected ALGS patients
  • Figure 3: Frequency of symptoms
  • Figure 4: Total cases of Alagille syndrome in 7MM (2017-2030)
  • Figure 5: Total prevalent cases of Alagille syndrome in the United States (2017-2030)
  • Figure 6: Gender-specific cases of Alagille syndrome in the US (2017-2030)
  • Figure 7: Age-specific cases of Alagille syndrome in the US (2017-2030)
  • Figure 8: Total prevalent cases of Alagille syndrome in Germany (2017-2030)
  • Figure 9: Gender-specific cases of Alagille syndrome in Germany (2017-2030)
  • Figure 10: Age-specific cases of Alagille syndrome in Germany (2017-2030)
  • Figure 11: Total prevalent cases of Alagille syndrome in France (2017-2030)
  • Figure 12: Gender-specific cases of Alagille syndrome in France (2017-2030)
  • Figure 13: Age-specific cases of Alagille syndrome in France (2017-2030)
  • Figure 14: Total prevalent cases of Alagille syndrome in Italy (2017-2030)
  • Figure 15: Gender-specific cases of Alagille syndrome in Italy (2017-2030)
  • Figure 16: Age-specific cases of Alagille syndrome in Italy (2017-2030)
  • Figure 17: Total prevalent cases of Alagille syndrome in Spain (2017-2030)
  • Figure 18: Gender-specific cases of Alagille syndrome in Spain (2017-2030)
  • Figure 19: Age-specific cases of Alagille syndrome in Spain (2017-2030)
  • Figure 20: Total prevalent cases of Alagille syndrome in the United Kingdom (2017-2030)
  • Figure 21: Gender-specific cases of Alagille syndrome in the UK (2017-2030)
  • Figure 22: Age-specific cases of Alagille syndrome in the UK (2017-2030)
  • Figure 23: Total prevalent cases of Alagille syndrome in Japan (2017-2030)
  • Figure 24: Gender-specific cases of Alagille syndrome in Japan (2017-2030)
  • Figure 25: Age-specific cases of Alagille syndrome in Japan (2017-2030)
目次
Product Code: DIEI0883

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

Alagille syndrome Disease Understanding

Alagille syndrome is a genetic disorder that is caused by a mutation in either JAG1 or NOTCH 2 gene. These genes are located on chromosomes 20 and 1, respectively; this mutation is an autosomal dominant mutation with a large section of the population acquiring it de novo. It is a rare disease with an incidence of 1 in 30,000 and is characterized by high phenotypic variability. Alagille syndrome is generally associated with cholestasis, jaundice, pruritus, congenital heart defects, kidney defects, bone disorders like butterfly vertebrae, among others; a large number of patients require liver transplantation due to complications.

Many unmet needs are associated with this disorder; this disease is difficult to diagnose due to the unavailability of a reliable biomarker. Moreover, the current treatment available is symptomatic and do not address the underlying cause of the disease. Therefore, it becomes imperative that such needs are fulfilled to have an efficient and reliable method to treat this disease.

Alagille syndrome Epidemiology

The Alagille syndrome epidemiology division provides insights about the historical and current patient pool, along with the forecasted trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the our report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The total prevalent cases of Alagille syndrome patients are increasing in 7MM during the study period, i.e. 2017-2030.

The disease epidemiology covered in the report provides historical as well as forecasted Alagille syndrome symptoms epidemiology segmented as the Total Prevalent cases of Alagille syndrome, Gender-specific cases of Alagille syndrome, Age-specific cases of Alagille syndrome. The report includes the prevalent scenario of Alagille syndrome symptoms in 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.

Country-wise - Alagille syndrome Epidemiology

The epidemiology segment also provides the Alagille syndrome epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The total prevalent cases of Alagille syndrome associated in 7MM countries were 17,486 in 2017.

  • As per the estimates, the United States has the largest prevalent population of Alagille syndrome.
  • Among the EU5 countries, Germany had the highest prevalent cases of Alagille syndrome, followed by France. On the other hand, Spain had the lowest prevalent cases with 1,469 cases in 2017.

Scope of the Report:

  • The Alagille syndrome report covers a detailed overview explaining its causes, symptoms, and classification, pathophysiology, diagnosis, and treatment patterns.
  • The Alagille syndrome Epidemiology Report and Model provide an overview of the risk factors and global trends of Alagille syndrome in the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan).
  • The report provides insight into the historical and forecasted patient pool of Alagille syndrome in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan.
  • The report helps to recognize the growth opportunities in the 7MM concerning the patient population.
  • The report assesses the disease risk and burden and highlights the unmet needs of Alagille syndrome.
  • The report provides the segmentation of the Alagille syndrome epidemiology by Prevalent Cases of Alagille syndrome in 7MM.
  • The report provides the segmentation of the Alagille syndrome epidemiology by Gender-specific Prevalent Cases of Alagille syndrome in 7MM.
  • The report provides the segmentation of the Alagille syndrome epidemiology by Age-specific Prevalent Cases of Alagille syndrome in 7MM.

Report Highlights:

  • 11-year Forecast of Alagille syndrome epidemiology
  • 7MM Coverage
  • Total Prevalent Cases of Alagille syndrome
  • Prevalent Cases according to segmentation: Gender-specific cases of Alagille syndrome
  • Prevalent Cases according to segmentation: Age-specific cases of Alagille syndrome

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 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 Alagille syndrome?
  • What are the key findings pertaining to the Alagille syndrome epidemiology across 7MM and which country will have the highest number of patients during the forecast period (2017-2030)?
  • What would be the total number of patients of Alagille syndrome across the 7MM during the forecast period (2017-2030)?
  • Among the EU5 countries, which country will have the highest number of patients during the forecast period (2017-2030)?
  • At what CAGR the patient population is expected to grow in 7MM during the forecast period (2017-2030)?
  • What are the disease risk, burden, and unmet needs of the Alagille syndrome?
  • What are the currently available treatments of Alagille syndrome?

Reasons to buy:

The Alagille syndrome epidemiology report will allow the user to -

  • Develop business strategies by understanding the trends shaping and driving the global Alagille syndrome market
  • Quantify patient populations in the global Alagille syndrome market to improve product design, pricing, and launch plans
  • Organize sales and marketing efforts by identifying the age groups and sex that present the best opportunities for Alagille syndrome therapeutics in each of the markets covered
  • Understand the magnitude of Alagille syndrome population by its Prevalence cases
  • Understand the magnitude of Alagille syndrome population by its Gender-specific cases
  • Understand the magnitude of Alagille syndrome population by its Age-specific cases
  • The Alagille syndrome epidemiology report and model were written and developed by Masters and PhD level epidemiologists
  • The Alagille syndrome Epidemiology Model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based on 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.

Alagille syndrome is caused by a mutation in JAG1 or Notch2 gene. Almost 90% of cases are due to mutations in JAG1 (20p12), an additional 5-7% are due to deletions incorporating JAG1, and about 1% is due to mutations in NOTCH2 (1p13). ALGS may be referred to as type 1 (JAG1-associated) or type 2 (NOTCH2-associated).

Wathen et al. did a sequential screening of 247 clinically well-defined patients and found that JAG1 mutations were there in 232 out of 247 patients studied, and 83 of the mutations were novel. This increase in the mutation rate was accomplished by combining rigorous clinical phenotyping, with a combination of mutation detection techniques, including fluorescence in situ hybridization (FISH), genomic and cDNA sequencing, and quantitative PCR. Eighty-five percent of patients have bile duct paucity, 96% have chronic cholestasis, 97% have a cardiac murmur, 51% have vertebral anomalies, 96% have facies, and 40% have renal disease

Table of Contents

1 Key Insights

2 Executive Summary

3 Organizations

4 Epidemiology and market methodology

5 Alagille syndrome: market overview at a glance

  • 5.1 Total market share (%) distribution of Alagille syndrome in 2017
  • 5.2 Total market share (%) distribution of Alagille syndrome in 2030

6 Alagille syndrome: market overview at a glance

  • 6.1 Introduction
  • 6.2 Causes
  • 6.3 Signs and symptoms
  • 6.4 Clinical presentation
  • 6.5 Pathophysiology
  • 6.6 Prognosis
  • 6.7 Diagnosis

Diagnostic criteria

Differential diagnosis

7 Epidemiology and patient population

  • 7.1 Key findings
  • 7.2 Assumptions and rationale
  • 7.3 Total cases of Alagille syndrome in 7mm
  • 7.4 United States
    • 7.4.1 Total Prevalent cases of Alagille Syndrome in the United States
    • 7.4.2 Gender-specific cases of Alagille Syndrome in the United States
    • 7.4.3 Age-specific cases of Alagille Syndrome in the United States
  • 7.1 EU5 countries
  • 7.2 Germany
    • 7.2.1 Total Prevalent cases of Alagille Syndrome in Germany
    • 7.2.2 Gender-specific cases of Alagille Syndrome in Germany
    • 7.2.3 Age-specific cases of Alagille Syndrome in Germany
  • 7.3 France
    • 7.3.1 Total Prevalent cases of Alagille Syndrome in France
    • 7.3.2 Gender-specific cases of Alagille Syndrome in France
    • 7.3.3 Age-specific cases of Alagille Syndrome France
  • 7.4 Italy
    • 7.4.1 Total Prevalent cases of Alagille Syndrome in Italy
    • 7.4.2 Gender-specific cases of Alagille Syndrome in Italy
    • 7.4.3 Age-specific cases of Alagille Syndrome in Italy
  • 7.5 Spain
    • 7.5.1 Total Prevalent cases of Alagille Syndrome in Spain
    • 7.5.2 Gender-specific cases of Alagille Syndrome in Spain
    • 7.5.3 Age-specific cases of Alagille Syndrome Spain
  • 7.6 UK
    • 7.6.1 Total Prevalent cases of Alagille Syndrome in the United Kingdom
    • 7.6.2 Gender-specific cases of Alagille Syndrome in the United Kingdom
    • 7.6.3 Age-specific cases of Alagille Syndrome in the United Kingdom
  • 7.7 Japan
    • 7.7.1 Total Prevalent cases of Alagille Syndrome in Japan
    • 7.7.2 Gender-specific cases of Alagille Syndrome in Japan
    • 7.7.3 Age-specific cases of Alagille Syndrome in Japan

8 KOL views

9 Appendix

10 Report methodology

11 DelveInsight capabilities

12 Disclaimer

13 About DelveInsight

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