市場調查報告書

精氨酸琥珀酸尿症(ASA):市場考慮因素,流行病,市場預測(-2030)

Argininosuccinic aciduria (ASA) - Market Insights, Epidemiology, and Market Forecast - 2030

出版商 DelveInsight Business Research LLP 商品編碼 968105
出版日期 內容資訊 英文 119 Pages
商品交期: 最快1-2個工作天內
價格
精氨酸琥珀酸尿症(ASA):市場考慮因素,流行病,市場預測(-2030) Argininosuccinic aciduria (ASA) - Market Insights, Epidemiology, and Market Forecast - 2030
出版日期: 2020年10月30日內容資訊: 英文 119 Pages
簡介

本報告詳細介紹了美國,五個歐洲國家(德國,西班牙,意大利,法國和英國)和七個主要日本國家的精氨酸琥珀酸尿症(ASA)市場,並提供了該病的背景和概述,未滿足的需求,每個國家的流行病學,治療方法等。

目錄

第1章重要見解

第2章精氨酸琥珀酸酯(ASA):市場概述

  • 2017年:ASA市場份額(%)分佈
  • 2030年:ASA市場份額(%)分佈

第3章執行摘要

第4章組織

第5章疾病概述:精氨酸琥珀酸尿症

  • 簡介
  • 體徵和症狀
  • 臨床特徵
  • 病理生理學
  • 診斷

第6章流行病學和患者人群

  • 主要發現
  • 7個主要國家的精氨酸琥珀酸尿症病例總數
  • 先決條件和理由:7個主要國家
  • 美國
    • 案件總數
    • 早發和晚發病例
  • 德國
    • 案件總數
    • 早發和晚發病例
  • 法國
  • 意大利
  • 西班牙
  • 英國
    • 案件總數
    • 早發和晚發病例
  • 日本

第7章治療和管理

第8章未滿足的需求

第9章主要衝突

第10章非處方藥

  • 聯苯:地平線療法
    • 產品說明
    • 監管里程碑
    • 其他發展活動
    • 安全性和有效性
    • 產品簡介
  • Ravicti:地平線療法
    • 臨床發展

第11章新療法

  • ACER-001:宏cer療法
    • 產品說明
    • 其他發展活動
    • 臨床發展
    • 安全性和有效性
    • 產品簡介
  • 基於體內外泌體的治療:Evox治療學

第12章精氨酸琥珀酸酯:七個主要市場的分析

  • 主要發現
  • 7個主要國家/地區的市場規模

第13章市場前景

  • 美國市場規模
    • 總市場規模
    • 市場規模:按處理方法
  • 德國市場規模
    • 市場總規模
    • 市場規模:按治療方法
  • 法國市場規模
  • 意大利市場規模
  • 西班牙市場規模
  • 英國市場規模
    • 市場總規模
    • 市場規模:按處理方法
  • 日本市場規模

第14章市場推廣者

第15章市場壁壘

第16章市場准入

第17章SWOT分析

第18章案例研究

  • 精氨酸琥珀酸酯缺乏症患者首次報告帕金森綜合症
  • 新生兒篩查中忽略了精氨酸琥珀酸酯缺乏症
  • 青少年流行性感冒感染引起的遲發性精氨酸琥珀酸尿症與高氨血症相關:病例報告

第19章KOL的觀點

第20章KOL的觀點

第21章參考

第22章附錄

  • 報告研究方法

第23章Delve Insight服務內容

第24章免責聲明

關於第25章Delve Insight

目錄
Product Code: DIMI1050

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

The argininosuccinic aciduria (ASA) market report provides current treatment practices, emerging drugs, and their market share of the individual therapies, current and forecasted argininosuccinic aciduria (ASA) market Size from 2017 to 2030, segmented by seven major markets. The report also covers the current treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geographies Covered

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

Study Period: 2017-2030

Argininosuccinic Aciduria (ASA) Disease Understanding and Treatment Algorithm

Argininosuccinic Aciduria (ASA) Overview

Argininosuccinic aciduria (ASA) is one of the inherited disorders belonging to 'urea cycle disorders,' which causes 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 of acute metabolic decompensations with hyperammonemia involves discontinuing oral protein intake, supplementing oral intake with intravenous lipids and/or glucose, and the use of intravenous arginine and nitrogen-scavenging therapy. Dietary restriction of protein and dietary supplementation with arginine are the mainstays in long-term management. Orthotopic liver transplantation (OLT) is best considered only in patients with recurrent hyperammonemia or metabolic decompensations resistant to conventional medical therapy.

Ammonia scavengers may be required in those who have metabolic decompensations. Recent evidence also suggests that ASL is required to synthesize nitric oxide (NO) and that NO deficiency in this disorder may lead to hypertension. Various nitrogen scavengers have been approved to treat urea cycle disorders, including sodium benzoate, Buphenyl (Ammonaps in Europe), and Ravicti. Liver transplant should be an option for ASA patients to prevent further neurologic deterioration and improve their quality of life.

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) Diagnosis and Treatment

It covers the details of conventional and current medical therapies and diagnosis available in the argininosuccinic aciduria (ASA) market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.

The DelveInsight Argininosuccinic Aciduria (ASA) market report gives a thorough understanding of Argininosuccinic aciduria (ASA) by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides treatment algorithms and treatment guidelines for Argininosuccinic Aciduria (ASA) in the US, Europe, and Japan.

Argininosuccinic Aciduria (ASA) Epidemiology

The persistent 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 and forecasted argininosuccinic aciduria (ASA) epidemiology segmented as the total incident cases of argininosuccinic aciduria (ASA) and 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) in 7MM countries was estimated to be 393 in 2017.

Argininosuccinic Aciduria (ASA) Drug Chapters

The drug chapter segment of the argininosuccinic aciduria (ASA) report encloses the detailed analysis of argininosuccinic aciduria (ASA) marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the argininosuccinic aciduria (ASA) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Treatment of ASA may require the coordinated efforts of a team of specialists ranging from pediatricians, geneticists, dieticians, and physicians who are familiar with metabolic disorders to occupational, speech-language, and physical therapists. A comprehensive approach to treatment is needed to treat children with wide disabilities. Genetic counseling is also recommended for affected individuals and their families. Furthermore, treatment recommendations may be separated into two scenarios: treatment for rapid control of hyperammonemia during metabolic decompensations and interval therapy to prevent the primary manifestations and long-term complications.

Products detail in the report…

Argininosuccinic Aciduria (ASA) Emerging Drugs

ACER-001 is a proprietary, fully taste-masked, immediate-release formulation of sodium phenylbutyrate (NaPB) developed using a microencapsulation process. NaPB helps control blood ammonia levels in conjunction with a restricted diet for people with UCDs. For the treatment of UCDs, NaPB is a prodrug that metabolizes quickly to become phenylacetate that binds to glutamine via acetylation to form phenylacetylglutamine. Phenylacetylglutamine is similar to urea; it contains two moles of nitrogen and is excreted by the kidneys. Through this process, NaPB is an alternate way to excrete excessive nitrogen created by the ineffective processing of ammonia during the urea cycle.

Products detail in the report…

Argininosuccinic Aciduria (ASA) Market Outlook

The argininosuccinic aciduria (ASA) market outlook of the report builds the detailed comprehension of the historical, current, and forecasted argininosuccinic aciduria (ASA) market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand of better technology.

This segment gives a thorough detail of Argininosuccinic Aciduria (ASA) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, Argininosuccinic Aciduria (ASA) market in 7MM is expected to change in the study period 2017-2030.

The management and diagnosis of ASA have not advanced significantly in the past decade, but given recent advances in understanding the timing and potential pathogenesis of ASA, it is hopeful that the next decade will bring early diagnostics and novel therapeutics.

Key Findings

This section includes a glimpse of the argininosuccinic aciduria (ASA) market in the 7MM. The market size of argininosuccinic aciduria (ASA) in the seven major markets was found to be USD 40.3 million in 2017.

The United States Market Outlook

This section provides the total Argininosuccinic Aciduria (ASA) market size and market size by therapies in the United States.

The United States accounts for the highest market size of Argininosuccinic Aciduria (ASA) in comparison to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

The treatment of ASA aims to prevent the formation of excessive ammonia or the removal of excess ammonia during a hyperammonemic episode. Treatment recommendations may be separated into two scenarios, i.e., treatment for rapid control of hyperammonemia during metabolic decompensations and interval therapy, to prevent the primary manifestations and long-term complications.

Dietary restrictions in individuals with ASA are aimed at limiting the amount of protein intake to avoid the development of excess ammonia. However, enough protein must be taken in by an affected infant to ensure proper growth.

In addition to dietary restrictions and supplements, individuals with ASA are treated by medications that stimulate the removal of nitrogen from the body. These medications provide an alternative method to the urea cycle in converting and removing nitrogen waste; this is known as alternative pathway therapy or nitrogen scavenging therapy and includes sodium benzoate, sodium phenylbutyrate glycerol tri-phenylbutyrate, and others.

Oral nitrogen-scavenging therapy, patients who have had metabolic decompensations or episodes of elevated ammonia despite being on a protein-restricted diet and arginine base supplementation are candidates for oral nitrogen-scavenging therapy, in which sodium benzoate and sodium phenylbutyrate stimulate the excretion of nitrogen in the form of hippuric acid and phenylacetylglutamine, respectively.

There is no cure for urea cycle disorders other than a liver transplant. However, a liver transplant is limited by donor availability and patient eligibility. It is also associated with significant risks and complications, including perioperative morbidity and mortality, liver rejection, and vulnerability to infection due to lifelong immunosuppressant medication.

For the prevention of secondary complications, recommendations for salt restriction, and the use of antihypertensive therapy in those with elevated blood pressures are not different from those for the general population. Potassium supplementation must be considered in those with hypokalemia.

EU-5 Countries: Market Outlook

The total argininosuccinic aciduria (ASA) market size and market size by therapies in EU-5 are also mentioned.

Japan Market Outlook

The total argininosuccinic aciduria (ASA) market size and market size by therapies in Japan are also mentioned.

Argininosuccinic Aciduria (ASA) Drugs Uptake

This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2017-2030. The analysis covers argininosuccinic aciduria (ASA) market uptake by drugs, patient uptake by therapies, and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

Argininosuccinic Aciduria (ASA) Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II and Phase III stages. It also analyses argininosuccinic aciduria (ASA) key players involved in developing targeted therapeutics.

Major players include Acer Therapeutics, Horizon Therapeutics, and Evox Therapeutics, whose key products are expected to get launched in the US market by 20XX.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for argininosuccinic aciduria (ASA) emerging therapies.

KOL- Views

To keep up with current market trends, we take KOLs and SME's opinion working in the argininosuccinic aciduria (ASA) domain through primary research to fill the data gaps and validate our secondary research. Their opinions help to understand and validate current and emerging therapies treatment patterns or argininosuccinic aciduria (ASA) market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive & Market Intelligence analysis of the Argininosuccinic Aciduria (ASA) Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report:

  • The report covers the descriptive overview of argininosuccinic aciduria (ASA), explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
  • Comprehensive insight has been provided into the argininosuccinic aciduria (ASA) epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for argininosuccinic aciduria (ASA) is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
  • A detailed review of the argininosuccinic aciduria (ASA) market - historical and forecasted - is included in the report, covering drug outreach in the 7MM
  • The report provides an edge while developing business strategies by understanding trends shaping and driving the global argininosuccinic aciduria (ASA) market.

Report Highlights:

  • In the coming years, argininosuccinic aciduria (ASA) market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence argininosuccinic aciduria (ASA) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Major players are involved in developing therapies for Argininosuccinic Aciduria (ASA). The launch of emerging therapies will significantly impact the Argininosuccinic Aciduria (ASA) market.
  • A better understanding of disease pathogenesis will also contribute to developing novel therapeutics for argininosuccinic aciduria (ASA).
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key competitors, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

Argininosuccinic Aciduria (ASA) Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Argininosuccinic Aciduria (ASA) Pipeline Analysis
  • Argininosuccinic Aciduria (ASA) Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Argininosuccinic Aciduria (ASA) Report Key Strengths

  • 11-years Forecast
  • 7MM Coverage
  • Argininosuccinic Aciduria (ASA) Epidemiology Segmentation
  • Key Competitors
  • Highly Analyzed Market
  • Drugs Uptake

Argininosuccinic Aciduria (ASA) Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions:

Market Insights:

  • What were the argininosuccinic aciduria (ASA) Market share (%) distribution in 2017, and how it would look like in 2030?
  • What would be the argininosuccinic aciduria (ASA) total market Size and market Size by therapies across the 7MM during the forecast period (2017-2030)?
  • What are the key findings pertaining to the market across 7MM, and which country will have the largest argininosuccinic aciduria (ASA) market Size during the forecast period (2017-2030)?
  • At what CAGR, the argininosuccinic aciduria (ASA) market is expected to grow in 7MM during the forecast period (2017-2030)?
  • What would be the argininosuccinic aciduria (ASA) market outlook across the 7MM during the forecast period (2017-2030)?
  • What would be the argininosuccinic aciduria (ASA) market growth till 2030, and what will be the resultant market Size in the year 2030?
  • How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

  • What are the disease risk, burden, and unmet needs of the Argininosuccinic Aciduria (ASA)?
  • What is the historical Argininosuccinic Aciduria (ASA) patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What would be the forecasted patient pool of argininosuccinic aciduria (ASA) in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to argininosuccinic aciduria (ASA)?
  • Out of all 7MM countries, which country would have the highest incident population of argininosuccinic aciduria (ASA) during the forecast period (2017-2030)?
  • At what CAGR the population is expected to grow in the 7MM during the forecast period (2017-2030)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

  • What are the current options for the treatment of argininosuccinic aciduria (ASA)?
  • What are the current treatment guidelines for treating argininosuccinic aciduria (ASA) in the USA, Europe, and Japan?
  • What are the argininosuccinic aciduria (ASA) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing therapies for the treatment of argininosuccinic aciduria (ASA)?
  • How many therapies are developed by each company for the treatment of argininosuccinic aciduria (ASA)?
  • How many emerging therapies are in the mid-stage and late stages of development for the treatment of argininosuccinic aciduria (ASA)?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the argininosuccinic aciduria (ASA) therapies?
  • What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for argininosuccinic aciduria (ASA) and their status?
  • What are the key designations that have been granted for the emerging therapies argininosuccinic aciduria (ASA)?
  • What are the global historical and forecasted market of argininosuccinic aciduria (ASA)?

Reasons to buy:

  • The report will help in developing business strategies by understanding trends shaping and driving the argininosuccinic aciduria (ASA) market.
  • To understand the future market competition in the argininosuccinic aciduria (ASA) market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for argininosuccinic aciduria (ASA) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for argininosuccinic aciduria (ASA) market.
  • To understand the future market competition in the argininosuccinic aciduria (ASA) market.

Table of Contents

1. Key Insights

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

  • 2.1. Market Share (%) Distribution of ASA in 2017
  • 2.2. Market 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. Treatment and Management

8. Unmet Needs

9. Key Cross Competition

10. Marketed Drugs

  • 10.1. Buphenyl: Horizon Therapeutics
    • 10.1.1. Product Description
    • 10.1.2. Regulatory milestones
    • 10.1.3. Other Developmental Activities
    • 10.1.4. Safety and Efficacy
    • 10.1.5. Product Profile
  • 10.2. Ravicti: Horizon Therapeutics
    • 10.2.1. Product Description
    • 10.2.2. Regulatory milestones
    • 10.2.3. Other Developmental Activities
    • 10.2.4. Clinical Development
    • 10.2.5. Safety and Efficacy
    • 10.2.6. Product Profile

11. Emerging Therapies

  • 11.1. ACER-001: Acer Therapeutics
    • 11.1.1. Product Description
    • 11.1.2. Other developmental activities
    • 11.1.3. Clinical Development
    • 11.1.4. Safety and Efficacy
    • 11.1.5. Product Profile
  • 11.2. In vivo exosome-based therapy: Evox Therapeutics
    • 11.2.1. Product Description
    • 11.2.2. Other developmental activities
    • 11.2.3. Safety and Efficacy
    • 11.2.4. Product Profile

12. Argininosuccinic aciduria: Seven Major Market Analysis

  • 12.1. Key Findings
  • 12.2. Market Size of Argininosuccinic aciduria in the 7MM

13. Market Outlook

  • 13.1. United States Market Size
    • 13.1.1. Total market size of Argininosuccinic aciduria in the United States
    • 13.1.2. Market size of Argininosuccinic aciduria by Therapies in the US
  • 13.2. Germany Market Size
    • 13.2.1. Total market size of Argininosuccinic aciduria in Germany
    • 13.2.2. Market size of Argininosuccinic aciduria by Therapies in Germany
  • 13.3. France Market Size
    • 13.3.1. Total market size of Argininosuccinic aciduria in France
    • 13.3.2. Market size of Argininosuccinic aciduria by Therapies in France
  • 13.4. Italy Market Size
    • 13.4.1. Total market size of Argininosuccinic aciduria in Italy
    • 13.4.2. Market size of Argininosuccinic aciduria by Therapies in Italy
  • 13.5. Spain Market Size
    • 13.5.1. Total market size of Argininosuccinic aciduria in Spain
    • 13.5.2. Market size of Argininosuccinic aciduria by Therapies in Spain
  • 13.6. UK Market Size
    • 13.6.1. Total market size of Argininosuccinic aciduria in the UK
    • 13.6.2. Market size of Argininosuccinic aciduria by Therapies in the UK
  • 13.7. Japan Market Size
    • 13.7.1. Total market size of Argininosuccinic aciduria in Japan
    • 13.7.2. Market size of Argininosuccinic aciduria by Therapies in Japan

14. Market Drivers

15. Market Barriers

16. Market Access

17. SWOT Analysis

18. Case studies

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

19. KOL Views

20. KOL views

21. Bibliography

22. Appendix

  • 22.1. Report Methodology

23. DelveInsight Capabilities

24. Disclaimer

25. About DelveInsight

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)
  • Table 19: Recommended Surveillance for Individuals with Argininosuccinate Lyase Deficiency
  • Table 20: Key Competitors
  • Table 21: Ravicti, Clinical Trial Description, 2020
  • Table 22: Market Size of Argininosuccinic aciduria in 7MM in USD Million (2017-2030)
  • Table 23: Total Market Size of Argininosuccinic aciduria in the US in USD Million (2017-2030)
  • Table 24: The US Market Size of ASA by Therapies, in USD Million (2017-2030)
  • Table 25: Total Market Size of Argininosuccinic aciduria in Germany in USD Million (2017-2030)
  • Table 26: Germany Market Size of ASA by Therapies, in USD Million (2017-2030)
  • Table 27: Total Market Size of Argininosuccinic aciduria in France in USD Million (2017-2030)
  • Table 28: France Market Size of ASA by Therapies, in USD Million (2017-2030)
  • Table 29: Total Market Size of Argininosuccinic aciduria in Italy in USD Million (2017-2030)
  • Table 30: Italy Market Size of ASA by Therapies, in USD Million (2017-2030)
  • Table 31: Total Market Size of Argininosuccinic aciduria in Spain in USD Million (2017-2030)
  • Table 32: Spain Market Size of ASA by Therapies, in USD Million (2017-2030)
  • Table 33: Total Market Size of Argininosuccinic aciduria in the UK in USD Million (2017-2030)
  • Table 34: The UK Market Size of ASA by Therapies, in USD Million (2017-2030)
  • Table 35: Total Market Size of Argininosuccinic aciduria in Japan in USD Million (2017-2030)
  • Table 36:Japan Market Size of ASA by Therapies, in USD Million (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 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)
  • Figure 20: Unmet Needs
  • Figure 21: Market Size of Argininosuccinic aciduria in USD Million (2017-2030)
  • Figure 22: Total Market Size of Argininosuccinic aciduria in the US, USD Millions (2017-2030)
  • Figure 23: The US Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 24: Total Market Size of Argininosuccinic aciduria in Germany, USD Millions (2017-2030)
  • Figure 25: Germany Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 26: Total Market Size of Argininosuccinic aciduria in France, USD Millions (2017-2030)
  • Figure 27: France Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 28: Total Market Size of Argininosuccinic aciduria in Italy, USD Millions (2017-2030)
  • Figure 29: Italy Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 30: Total Market Size of Argininosuccinic aciduria in Spain, USD Millions (2017-2030)
  • Figure 31: Spain Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 32: Total Market Size of Argininosuccinic aciduria in the UK, USD Millions (2017-2030)
  • Figure 33: The UK Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 34: Total Market Size of Argininosuccinic aciduria in Japan, USD Millions (2017-2030)
  • Figure 35: Japan Market Size of ASA by Therapies, USD Millions (2017-2030)
  • Figure 36: Market Drivers
  • Figure 37:Market Barriers