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アルギニノコハク酸尿症(ASA):2030年までの疫学予測

Argininosuccinic aciduria (ASA) - Epidemiology Forecast to 2030

出版日: 受注後作成 | 発行: DelveInsight Business Research LLP | ページ情報: 英文 63 Pages | 納期: 2~10営業日

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アルギニノコハク酸尿症(ASA):2030年までの疫学予測
出版日: 受注後作成
発行: DelveInsight Business Research LLP
ページ情報: 英文 63 Pages
納期: 2~10営業日
  • 全表示
  • 概要
  • 図表
  • 目次
概要

アルギニノコハク酸尿症(ASA)は、「尿素回路異常」に属する遺伝性疾患の1つです。ASAの患者は、過剰な窒素を取り除くために必要な肝酵素の1つである「アルギニノコハク酸リアーゼ」を欠いており、過剰な窒素がアンモニアの形で体内に蓄積し、特に脳で高レベルの毒性を示す可能性があります。

ASAの症状は通常生後数日で現れ、無気力(エネルギー不足)、嘔吐、食欲不振、発作(発作)、昏睡などのほか、精神遅滞も生じ、長期にわたる衰弱によって生命が脅かされます。治療は、小児科医、神経科医、遺伝学者、栄養士、代謝障害に精通している医師が協力しながら、包括的に進める必要があります。また発達障害のある子供の治療には、作業療法士、言語療法士、理学療法士の支援が必要です。

当レポートは、米国、ドイツ、フランス、イタリア、スペイン、英国、日本の主要7ヶ国のアルギニノコハク酸尿症(ASA)の疫学と関連市場について調査したもので、疾患の概要、疫学と患者数、SWOT分析、ケーススタディ、主な専門家の見解などの情報を提供しています。

目次

第1章 主な調査結果

第2章 疫学の概要

第3章 エグゼクティブサマリー

第4章 組織

第5章 疾患の概要

  • イントロダクション
  • 徴候と症状
  • 臨床的特徴
  • 病態生理学
  • 診断

第6章 疫学と患者数

  • 主な調査結果
  • 主要7ヶ国における新規症例数
  • 前提と根拠
  • 米国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
  • 日本

第7章 SWOT分析

第8章 ケーススタディ

第9章 主な専門家の見解

第10章 参考文献

第11章 付録

第12章 DelveInsightのサービス内容

第13章 免責事項

第14章 DelveInsightについて

図表

List of Tables

  • Table 1: Upper Limits of Normal Plasma Ammonia Concentration by Age
  • Table 2: Age-Related Plasma Amino Acid Concentrations in ASL Deficiency
  • Table 3: Proposed monitoring for patients with argininosuccinic aciduria (ASA)
  • Table 4: Total Incident Population of ASA in 7MM (2017-2030)
  • Table 5: Total Incident Cases of ASA in the US (2017-2030)
  • Table 6: Early-onset and Late-onset Cases of ASA in the US (2017-2030)
  • Table 7: Total Incident Cases of ASA in Germany (2017-2030)
  • Table 8: Early-onset and Late-onset Cases of ASA in Germany (2017-2030)
  • Table 9: Total Incident Cases of ASA in France (2017-2030)
  • Table 10: Early-onset and Late-onset Cases of ASA in France (2017-2030)
  • Table 11: Total Incident Cases of ASA in Italy (2017-2030)
  • Table 12: Early-onset and Late-onset Cases of ASA in Italy (2017-2030)
  • Table 13: Total Incident Cases of ASA in Spain (2017-2030)
  • Table 14: Early-onset and Late-onset Cases of ASA in Spain (2017-2030)
  • Table 15: Total Incident Cases of ASA in the UK (2017-2030)
  • Table 16: Early-onset and Late-onset Cases of ASA in the UK (2017-2030)
  • Table 17: Total Incident Cases of ASA in Japan (2017-2030)
  • Table 18: Early-onset and Late-onset Cases of ASA in Japan (2017-2030)

List of Figures

  • Figure 1: Systemic phenotype in Argininosuccinic aciduria
  • Figure 2: The Urea Cycle
  • Figure 3: Pathophysiological mechanisms in Argininosuccinic Aciduria
  • Figure 4: Stepwise diagnostic procedure for investigational hyperammonemia
  • Figure 5: Total Incident Population of ASA in the 7MM (2017-2030)
  • Figure 6: Total Incident Cases of ASA in the US (2017-2030)
  • Figure 7: Early-onset and Late-onset Cases of ASA in the US (2017-2030)
  • Figure 8: Total Incident Cases of ASA in Germany (2017-2030)
  • Figure 9: Early-onset and Late-onset Cases of ASA in Germany (2017-2030)
  • Figure 10: Total Incident Cases of ASA in France (2017-2030)
  • Figure 11: Early-onset and Late-onset Cases of ASA in France (2017-2030)
  • Figure 12: Total Incident Cases of ASA in Italy (2017-2030)
  • Figure 13: Early-onset and Late-onset Cases of ASA in Italy (2017-2030)
  • Figure 14: Total Incident Cases of ASA in Spain (2017-2030)
  • Figure 15: Early-onset and Late-onset Cases of ASA in Spain (2017-2030)
  • Figure 16: Total Incident Cases of ASA in the UK (2017-2030)
  • Figure 17: Early-onset and Late-onset Cases of ASA in the UK (2017-2030)
  • Figure 18: Total Incident Cases of ASA in Japan (2017-2030)
  • Figure 19: Early-onset and Late-onset Cases of ASA in Japan (2017-2030)
目次
Product Code: DIEI1111

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

Argininosuccinic Aciduria (ASA) Disease Understanding

Argininosuccinic Aciduria (ASA) Overview

Argininosuccinic aciduria (ASA) is one of the inherited disorders that belongs to 'urea cycle disorders,' which cause ammonia to accumulate in the blood. Patients with ASA lack 'argininosuccinate lyase,' one of the liver enzymes needed to get rid of excess nitrogen. In the absence of this enzyme, excess nitrogen accumulates in the body in the form of ammonia, which can be toxic at high levels, especially to the brain.

The disease's symptoms usually appear in the first few days of life and include lethargy (lack of energy), vomiting, loss of appetite, seizures (fits), and coma. It is a long-term debilitating and life-threatening disease that leads to mental retardation and is associated with poor overall survival.

ASA can manifest as either a severe neonatal-onset form with hyperammonemia within the first few days after birth or as a Late-onset form with episodic hyperammonemia and/or long-term complications that include liver dysfunction, neurocognitive deficits, and hypertension. Treatment may require the coordinated efforts of a team of specialists. Pediatricians, neurologists, geneticists, dieticians, and physicians familiar with metabolic disorders may need to work together to ensure a comprehensive approach to treatment. Occupational, speech-language, and physical therapists may be needed to treat children with developmental disabilities. Genetic counseling is recommended for affected individuals and their families.

Moreover, while acute hyperammonemia in ASA should continue to be treated with high dose intravenous arginine, in the context of chronic management, lower dose arginine in conjunction with nitrogen-scavenging therapy, especially in patients with elevated liver enzyme tests, should be considered.

Argininosuccinic Aciduria (ASA) Epidemiology

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

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted argininosuccinic aciduria (ASA) epidemiology segmented as the total incident cases of argininosuccinic aciduria (ASA), Early-onset and Late-onset cases of argininosuccinic aciduria. The report includes the incident scenario of argininosuccinic aciduria (ASA) in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.

Country Wise- Argininosuccinic Aciduria (ASA) Epidemiology

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

The total Incident population of argininosuccinic aciduria (ASA) associated in the 7MM countries estimated to be was 393 in 2017.

As per the estimates, the United States has the highest Incident population of Argininosuccinic Aciduria (ASA).

  • Among the European 5 countries, Germany had the highest incident population of ASA with 35 cases, followed by France and the United Kingdom. On the other hand, Spain had the lowest incident cases of ASA, i.e., 21 in 2017.

Scope of the Report:

  • Argininosuccinic aciduria (ASA) report covers a detailed overview explaining its causes, symptoms, classification, pathophysiology, diagnosis, and treatment patterns.
  • Argininosuccinic aciduria (ASA) Epidemiology Report and Model provide an overview of the risk factors and global trends of argininosuccinic aciduria (ASA) 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 argininosuccinic aciduria (ASA) 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 argininosuccinic aciduria (ASA).
  • The report provides the segmentation of the argininosuccinic aciduria (ASA) epidemiology by incident cases of argininosuccinic aciduria (ASA) in the 7MM.
  • The report provides the segmentation of the argininosuccinic aciduria (ASA) epidemiology by Early-onset and Late-onset cases of argininosuccinic aciduria and incident cases of argininosuccinic aciduria (ASA) in the 7MM.

Report Highlights:

  • 11-year Forecast of Argininosuccinic Aciduria (ASA) epidemiology
  • 7MM Coverage
  • Total Incident Cases of Argininosuccinic Aciduria (ASA)
  • Incident Cases according to segmentation: Etiology-specific Incidence of Argininosuccinic Aciduria (ASA)

KOL- Views

DelveInsight interviews KOLs and obtain 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 for the patient population pertaining to argininosuccinic aciduria (ASA)?
  • What are the key findings pertaining to the argininosuccinic aciduria (ASA) 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 argininosuccinic aciduria (ASA) 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 by in the 7MM during the forecast period (2017-2030)?
  • What are the disease risk, burden, and unmet needs of the argininosuccinic aciduria (ASA)?
  • What are the currently available treatments of argininosuccinic aciduria (ASA)?

Reasons to buy:

The Argininosuccinic Aciduria (ASA) Epidemiology report will allow the user to:

  • Develop business strategies by understanding the trends shaping and driving the global argininosuccinic aciduria (ASA) market.
  • Quantify patient populations in the global argininosuccinic aciduria (ASA) 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 argininosuccinic aciduria (ASA) therapeutics in each of the markets covered.
  • Understand the magnitude of the argininosuccinic aciduria (ASA) population by its Incidence cases.
  • Understand the magnitude of the argininosuccinic aciduria (ASA) population by its etiology-specific cases.
  • The argininosuccinic aciduria (ASA) epidemiology report and model were written and developed by Masters and Ph.D. level epidemiologists.
  • The argininosuccinic aciduria (ASA) 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 an 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

According to Rare Diseases Clinical Research Network (RDCRN), ASA is the second most common UCD with the occurrence of approximately 1 in 70,000. Furthermore, according to other literature review, the worldwide frequency of ASA is estimated to be 1-9/1,000,000.

Table of Contents

1. Key Insights

2. Argininosuccinic Aciduria (ASA): Epidemiology Overview at a Glance

  • 2.1. Patient Share (%) Distribution of ASA in 2017
  • 2.2. Patient Share (%) Distribution of ASA in 2030

3. Executive summary

4. Organizations

5. Disease Overview: Argininosuccinic aciduria

  • 5.1. Introduction
  • 5.2. Signs and Symptoms
  • 5.3. Clinical Characterization
  • 5.4. Pathophysiology
  • 5.5. Diagnosis

6. Epidemiology and Patient Population

  • 6.1. Key Findings
  • 6.2. Total Incident Population of Argininosuccinic Aciduria in 7MM
  • 6.3. Assumptions and Rationale: 7MM
  • 6.4. United States
    • 6.4.1. Total Incident cases of Argininosuccinic Aciduria in the US
    • 6.4.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in the US
  • 6.5. Germany
    • 6.5.1. Total Incident cases of Argininosuccinic Aciduria in Germany
    • 6.5.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in Germany
  • 6.6. France
    • 6.6.1. Total Incident cases of Argininosuccinic Aciduria in France
    • 6.6.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in France
  • 6.7. Italy
    • 6.7.1. Total Incident cases of Argininosuccinic Aciduria in Italy
    • 6.7.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in Italy
  • 6.8. Spain
    • 6.8.1. Total Incident cases of Argininosuccinic Aciduria in Spain
    • 6.8.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in Spain
  • 6.9. UK
    • 6.9.1. Total Incident cases of Argininosuccinic Aciduria in the UK
    • 6.9.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in the UK
  • 6.10. Japan
    • 6.10.1. Total Incident cases of Argininosuccinic Aciduria in Japan
    • 6.10.2. Early-onset and Late-onset Cases of Argininosuccinic Aciduria in Japan

7. SWOT Analysis

8. Case studies

  • 8.1. First Reported Case of Parkinsonism in a Patient with Argininosuccinate Lyase Deficiency
  • 8.2. Argininosuccinic Acid Lyase Deficiency Missed by Newborn Screen
  • 8.3. Late-onset argininosuccinic aciduria associated with hyperammonemia triggered by influenza infection in an adolescent: A case report

9. KOL Views

10. Bibliography

11. Appendix

  • 11.1. Report Methodology

12. DelveInsight Capabilities

13. Disclaimer

14. About DelveInsight