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.
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.
• 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
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
• Patient Segmentation
• Disease Risk and Burden
• Risk of disease by the segmentation
• Factors driving growth in a specific patient population
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
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
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