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市場調查報告書

Farber氏病:流行病學洞察(至2030年的預測)

Farber's disease - Epidemiology Insight - 2030

出版商 DelveInsight Business Research LLP 商品編碼 966777
出版日期 按訂單生產 內容資訊 英文 66 Pages
商品交期: 10個工作天內
價格
Farber氏病:流行病學洞察(至2030年的預測) Farber's disease - Epidemiology Insight - 2030
出版日期: 按訂單生產內容資訊: 英文 66 Pages
簡介

本報告提供世界主要7個國家(美國,歐洲5個國家(德國,法國,義大利,西班牙,英國),日本)的Farber氏病的相關調查,流行病學及預測,各市場區隔的病例,診斷案例等資訊。

目錄

第1章 主要洞察

第2章 摘要整理

第3章 疾病的背景和概要

  • 概要
  • 徵兆與症狀
  • 病理學
  • 危險因素
  • 診斷

第4章 患者的治療過程

第5章 流行病學和患者人口

  • 流行病學的主要調查結果
  • 前提條件與理論性根據:主要7個國家
  • 流行病學方案:主要7個國家
    • 流行病學方案(2017-2030)
  • 美國流行病學
  • 歐洲5國流行病學
    • 德國流行病學
    • 法國流行病學
    • 義大利流行病學
    • 西班牙流行病學
    • 英國流行病學
  • 日本流行病學

第6章 治療流程,目前治療,及醫務

  • 治療和管理
  • 治療流程

第7章 KOL的見解

第8章 未滿足需求

第9章 附錄

  • 參考文件
  • 報告的調查手法

第10章 DelveInsight的服務內容

第11章 免責聲明

第12章 關於DelveInsight

目錄
Product Code: DIEI1052

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

Farber's disease: Disease Understanding

Farber's disease Overview

Farber's disease is a rare inherited condition involving the breakdown and use of fats in the body. In affected individuals, lipids accumulate abnormally in cells and tissues throughout the body, particularly around the joints. The deficiency of an enzyme is called ceramidase resulting in the harmful accumulation of certain chemicals in the body, which causes damage and inflammation.

Three classic signs occur in Farber lipogranulomatosis: a hoarse voice or a weak cry, small lumps of fat under the skin and in other tissues (lipogranulomas), and swollen and painful joints. Affected individuals may also have difficulty breathing, an enlarged liver and spleen (hepatosplenomegaly), and developmental delay.

Researchers have described seven types of Farber lipogranulomatosis based on their characteristic features: Type 1 is the most common, or classical, form of this condition and is associated with the classic signs of voice, skin, and joint problems that begin a few months after birth. Developmental delay and lung disease also commonly occur. Infants born with type 1 Farber lipogranulomatosis usually survive only into early childhood.

Types 2 and 3 generally have less severe signs and symptoms than the other types. Affected individuals have three classic signs and usually do not have developmental delays. Children with these types of Farber lipogranulomatosis typically live into mid- to late childhood.

Types 4 and 5 are associated with severe neurological problems. Type 4 usually causes life-threatening health problems beginning in infancy due to massive lipid deposits in the liver, spleen, lungs, and immune system tissues. Children with this type typically do not survive past their first year of life. Type 5 is characterized by a progressive decline in the brain and spinal cord (central nervous system) function, which causes paralysis of the arms and legs (quadriplegia), seizures, loss of speech, involuntary muscle jerks (myoclonus), and developmental delay. Children with type 5 Farber lipogranulomatosis survive into early childhood. Due to the extremely progressive and severe nature of the disease, current treatments focus on symptom management and enhancing an individual's quality of life rather than completely eliminating symptoms. Corticosteroids are commonly used to relieve pain associated with inflammation of the joints. Some individuals require surgical procedures to restore proper breathing, which is impaired by abnormal tissue growth in the airway. Additionally, some individuals undergo cosmetic surgery to remove nodules and abnormal growths of the facial area.

Farber's disease: Epidemiology

The Farber's disease 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 DelveInsight 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 Farber's disease epidemiology segmented as the total diagnosed prevalent cases of Farber's disease, gender-specific cases of Farber's disease, and type-specific cases of Farber's disease. The report includes the Prevalent scenario of Farber's disease 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 Farber's disease Epidemiology

The epidemiology segment also provides the Farber's disease epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The total prevalent population of Farber's disease in the 7MM countries was estimated to be 86 cases in 2017.

As per the estimates, in 2017, the United States had the highest prevalent population of Farber's disease. Among the EU5 countries, Germany had the highest prevalent population of Farber's disease with 8 cases, followed by France in 2017. On the other hand, Spain had the lowest prevalent population in 2017.

Scope of the Report:

  • Farber's disease report covers a detailed overview explaining its causes, symptoms, classification, pathophysiology, diagnosis, and treatment patterns
  • Farber's disease Epidemiology Report and Model provide an overview of the risk factors and global trends of Farber's disease 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 Farber's disease 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 with respect to the patient population
  • The report assesses the disease risk and burden and highlights the unmet needs of Farber's disease
  • The report provides the segmentation of the Farber's disease epidemiology by Prevalent Cases of Farber's disease in the 7MM
  • The report provides the segmentation of the Farber's disease epidemiology by Type-specific Prevalent Cases of Farber's disease in the 7MM

Report Highlights:

  • 11-year Forecast of Farber's disease epidemiology
  • 7MM Coverage
  • Total Prevalent Cases of Farber's disease
  • Type-specific Prevalence of Farber's disease
  • Gender-specific Prevalence of Farber's disease

KOL- Views

DelveInsight interview 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 with respect to the patient population pertaining to Farber's disease?
  • What are the key findings pertaining to the Farber's disease 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 Farber's disease 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, burdens, and unmet needs of Farber's disease?
  • What are the currently available treatments for Farber's disease?

Reasons to buy:

Farber's disease Epidemiology report will allow the user to:

  • Develop business strategies by understanding the trends shaping and driving the global Farber's disease market
  • Quantify patient populations in the global Farber's disease 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 Farber's disease therapeutics in each of the markets covered
  • Understand the magnitude of Farber's disease population by its Prevalence cases
  • Understand the magnitude of Farber's disease population by its Type-specific cases
  • The Farber's disease epidemiology report and model were written and developed by Masters and Ph.D. level epidemiologists
  • The Farber's disease Epidemiology Model develop 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

The Farber's disease Research Consortium in the USA, the European Farber's disease Registry in Europe, and the Japanese Farber's disease Registry (JBAR) have been formed to help with data collection and provide a platform for large research studies.

Table of Contents

1. Key Insights

2. Farber Disease: Epidemiology Overview at a Glance

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

3. Organizations

4. Executive Summary

5. Disease Overview: Farber Disease

  • 5.1. Introduction
  • 5.2. Classification
  • 5.3. Signs and Symptoms
  • 5.4. Clinical manifestations
  • 5.5. Pathophysiology
  • 5.6. Diagnosis
    • 5.6.1. Differential Diagnosis

6. Epidemiology and Patient Population

  • 6.1. Key Findings
  • 6.2. Total Diagnosed Prevalent Population of Farber Disease in 7MM

7. 7MM Epidemiology of Farber Disease

  • 7.1. Assumptions and Rationale
  • 7.2. United States
    • 7.2.1. Total Diagnosed Prevalent cases of Farber Disease in the US
    • 7.2.2. Gender-specific cases of Farber Disease in the US
    • 7.2.3. Type-specific cases of Farber Disease in the US
  • 7.3. Germany
    • 7.3.1. Total Diagnosed Prevalent cases of Farber Disease in Germany
    • 7.3.2. Gender-specific cases of Farber Disease in Germany
    • 7.3.3. Type-specific cases of Farber Disease in Germany
  • 7.4. France
    • 7.4.1. Total Diagnosed Prevalent cases of Farber Disease in France
    • 7.4.2. Gender-specific cases of Farber Disease in France
    • 7.4.3. Type-specific cases of Farber Disease in France
  • 7.5. Italy
    • 7.5.1. Total Diagnosed Prevalent cases of Farber Disease in Italy
    • 7.5.2. Gender-specific cases of Farber Disease in Italy
    • 7.5.3. Type-specific cases of Farber Disease in Italy
  • 7.6. Spain
    • 7.6.1. Total Diagnosed Prevalent cases of Farber Disease in Spain
    • 7.6.2. Gender-specific cases of Farber Disease in Spain
    • 7.6.3. Type-specific cases of Farber Disease in Spain
  • 7.7. The UK
    • 7.7.1. Total Diagnosed Prevalent cases of Farber Disease in the UK
    • 7.7.2. Gender-specific cases of Farber Disease in The UK
    • 7.7.3. Type-specific cases of Farber Disease in the UK
  • 7.8. Japan
    • 7.8.1. Total Diagnosed Prevalent cases of Farber Disease in Japan
    • 7.8.2. Gender-specific cases of Farber Disease in Japan
    • 7.8.3. Type-specific cases of Farber Disease in Japan

8. Bibliography

9. Appendix

  • 9.1. Report Methodology

10. DelveInsight Capabilities

11. Disclaimer

12. About DelveInsight

List of Tables

  • Table 1: Reported Mutations in ASAH1 that result in FD
  • Table 2: Recommended Evaluations Following Initial Diagnosis of an ASAH1-related Disorder: Farber Disease
  • Table 3: Recommended Evaluations Following Initial Diagnosis of an ASAH1-related Disorder: Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME)
  • Table 4: Total Diagnosed Prevalent Population of Farber Disease in the 7MM (2017-2030)
  • Table 5: Total Diagnosed Prevalent Cases of Farber Disease in the US (2017-2030)
  • Table 6: Gender-specific Cases of Farber Disease in the US (2017-2030)
  • Table 7: Type-specific Prevalent Cases of Farber Disease in the US (2017-2030)
  • Table 8: Total Diagnosed Prevalent Cases of Farber Disease in Germany (2017-2030)
  • Table 9: Gender-specific Prevalent Cases of Farber Disease in Germany (2017-2030)
  • Table 10: Type-specific Prevalent Cases of Farber Disease in Germany (2017-2030)
  • Table 11: Total Diagnosed Prevalent Cases of Farber Disease in France (2017-2030)
  • Table 12: Gender-specific Prevalent Cases of Farber Disease in France (2017-2030)
  • Table 13: Type-specific Prevalent Cases of Farber Disease in France (2017-2030)
  • Table 14: Total Diagnosed Prevalent Cases of Farber Disease in Italy (2017-2030)
  • Table 15: Gender-specific Prevalent Cases of Farber Disease in Italy (2017-2030)
  • Table 16: Type-specific Prevalent Cases of Farber Disease in Italy (2017-2030)
  • Table 17: Total Diagnosed Prevalent Cases of Farber Disease in Spain (2017-2030)
  • Table 18: Gender-specific Prevalent Cases of Farber Disease in Spain (2017-2030)
  • Table 19: Type-specific Prevalent Cases of Farber Disease in Spain (2017-2030)
  • Table 20: Total Diagnosed Prevalent Cases of Farber Disease in the UK (2017-2030)
  • Table 21: Gender-specific Prevalent Cases of Farber Disease in the UK (2017-2030)
  • Table 22: Type-specific Prevalent Cases of Farber Disease in the UK (2017-2030)
  • Table 23: Total Diagnosed Prevalent Cases of Farber Disease in Japan (2017-2030)
  • Table 24: Gender-specific Prevalent Cases of Farber Disease in Japan (2017-2030)
  • Table 25: Type-specific Prevalent Cases of Farber Disease in Japan (2017-2030)

List of Figures

  • Figure 1: Classification of Farber's Disease
  • Figure 2: Signs and Symptoms of Farber Disease
  • Figure 3: Clinical manifestations by Organ Type
  • Figure 4: Reaction schema of the hydrolysis of ceramide by acid ceramidase into sphingosine and free fatty acid
  • Figure 5: Diagnosis of Farber Disease
  • Figure 6: Total Diagnosed Prevalent Population of Farber Disease in 7MM (2017-2030)
  • Figure 7: Distribution of patients with Farber disease
  • Figure 8: Total Diagnosed Prevalent Cases of Farber Disease in the US (2017-2030)
  • Figure 9: Gender-specific Cases of Farber Disease in the US (2017-2030)
  • Figure 10: Type-specific Cases of Farber Disease in the US (2017-2030)
  • Figure 11: Total Diagnosed Prevalent Cases of Farber Disease in Germany (2017-2030)
  • Figure 12: Gender-specific Cases of Farber Disease in Germany (2017-2030)
  • Figure 13: Type-specific Cases of Farber Disease in Germany (2017-2030)
  • Figure 14: Total Diagnosed Prevalent Cases of Farber Disease in France (2017-2030)
  • Figure 15: Gender-specific Cases of Farber Disease in France (2017-2030)
  • Figure 16: Type-specific Cases of Farber Disease in France (2017-2030)
  • Figure 17: Total Diagnosed Prevalent Cases of Farber Disease in Italy (2017-2030)
  • Figure 18: Gender-specific Cases of Farber Disease in Italy (2017-2030)
  • Figure 19: Type-specific Cases of Farber Disease in Italy (2017-2030)
  • Figure 20: Total Diagnosed Prevalent Cases of Farber Disease in Spain (2017-2030)
  • Figure 21: Gender-specific Cases of Farber Disease in Spain (2017-2030)
  • Figure 22: Type-specific Cases of Farber Disease in Spain (2017-2030)
  • Figure 23: Total Diagnosed Prevalent Cases of Farber Disease in the UK (2017-2030)
  • Figure 24: Gender-specific Cases of Farber Disease in the UK (2017-2030)
  • Figure 25: Type-specific Cases of Farber Disease in the UK (2017-2030)
  • Figure 26: Total Diagnosed Prevalent Cases of Farber Disease in Japan (2017-2030)
  • Figure 27: Gender-specific Cases of Farber Disease in Japan (2017-2030)
  • Figure 28: Type-specific Cases of Farber Disease in Japan (2017-2030)