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Cystinuria- Epidemiology Forecast-2028

September 2019 | 87 pages | ID: C609EEF7781EN
DelveInsight

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DelveInsight’s ‘Cystinuria- Epidemiology Forecast-2028’ report deliver an in-depth understanding of the disease, historical & forecasted epidemiology trends of Cystinuria in the United States, EU5 (Germany, Spain, Italy, France and the United Kingdom), and Japan.

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan
Study Period: 2017–2028

Cystinuria - Disease Understanding

Cystinuria is an autosomal recessive disorder of renal tubular and intestinal transport of dibasic amino acids, which results in increased urinary excretion of cystine, and dibasic amino acids, such as ornithine, lysine and arginine (COLA). It may include either the presence of cystine stones (as a diagnosis) or increased urinary excretion of dibasic amino acids; along with mutations on both alleles of one of the two genes (SLC3A1; SLC7A9).

The clinical features are related to stone formation and might include nausea, flank pain, hematuria, recurrent urinary tract infections, and rarely acute or chronic renal failure. A variety of other symptoms and occurrences, including nausea, emesis, anorexia, and failure to thrive. It can be classified based on phenotypic features (Type 1 and Non-type 1 (type II, type III) and genotypic features TYPE A, TYPE B, TYPE AB). Males and females have a similar age of onset, wherein more male patients are affected in the first 3 years of life and males tend to develop more stones frequently. Type A and B genotypes apparently have similar phenotypes with similar rates of stone recurrence.

Cystinuria – Epidemiology

The Cystinuria epidemiology division provide insights about historical and current patient pool and forecasted trend for every 7 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.

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Prevalence of Cystinuria in 7MM, Gender-Specific Prevalence of Cystinuria in 7MM, Age-Specific Prevalence of Cystinuria, Diagnosed Prevalence of Cystinuria and Type-Specific Diagnosed Prevalence of Cystinuria), scenario of Cystinuria in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017–2028.

DelveInsight’s estimations suggests the total prevalent cases of Cystinuria in the 7MM was found to be approximately 85,876 in 2017. Among 7MM, United States has the highest prevalent population of Cystinuria with about 32,548 cases in 2017. In 2017, among EU-5 countries, Germany has the highest number of prevalent cases with approximately 11,831 cases, followed by France with approximately 9,602 cases. Spain has the least number of prevalent cases, while in Japan, the prevalent population of Cystinuria was found to be approximately 7,091.

Cystinuria is mainly divided in to three types based on the mutations in genes such as SLC3A1 and SLC7A9. Type A is caused by mutations in both SLC3A1 alleles, and type B is caused by mutations in both SLC7A9 alleles. Moreover, if mutations are found in single alleles of both genes, it is called as type AB, which is a rare and mild form. It has been shown that half of type A patients with cystinuria will form a stone in the first decade of life. There were total approximately 8,701 cases of Type A, 10,762 cases of Type B, and 3,206 cases of Type AB, in the US in 2017.

Cystinuria Report Insights
  • The report covers a descriptive overview of the Cystinuria, explaining its causes, risk factors, pathophysiology and diagnosis.
  • Comprehensive insight has been provided into the epidemiology Cystinuria in the 7MM countries covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
  • Assesses growth opportunities in the 7MM countries with respect to the patient population.
Cystinuria Report Key Strengths
  • 10 Year Forecast
  • 7MM Coverage
  • Epidemiology Segmentation by Type of Cystinuria
  • Total Patient Pool with Cystinuria
  • Diagnosed Patient Pool of Cystinuria
  • Gender-Specific Diagnosed Patient Pool of Cystinuria
  • Age-Specific Diagnosed Patient Pool of Cystinuria
  • Type-Specific Diagnosed Patient Pool of Cystinuria
Cystinuria Report Assessment
  • Current Treatment Practices
  • Unmet Needs
  • Detailed Pipeline Product Profiles
Key Benefits
  • Key assessments
  • Patient Segmentation
  • Disease Risk & Burden
  • Risk of disease by type
1. KEY INSIGHTS

2. CYSTINURIA: PATIENT OVERVIEW AT A GLANCE

2.1. Patient Share (%) Distribution of Cystinuria in 2017
2.2. Patient Share (%) Distribution of Cystinuria in 2028

3. DISEASE OVERVIEW: CYSTINURIA

3.1. Introduction
3.2. Classification
3.3. Clinical features
3.4. Genetics
3.5. Pathophysiology
3.6. Diagnosis

4. EPIDEMIOLOGY AND PATIENT POPULATION

4.1. Key Findings
4.2. 7MM Total Prevalent Patient Population of Cystinuria
4.3. Country Wise-Epidemiology of Cystinuria
4.4. United States
  4.4.1. Assumptions and Rationale
  4.4.2. Prevalent Cases of Cystinuria in the United States
  4.4.3. Gender-Specific Cases of Cystinuria in the United States
  4.4.4. Age-Specific Cases of Cystinuria in the United States
  4.4.5. Diagnosed Cases of Cystinuria in the United States
  4.4.6. Type-Specific Cases of Cystinuria in the United States
4.5. EU5
  4.5.1. Assumptions and Rationale
4.6. Germany
  4.6.1. Prevalent Cases of Cystinuria in Germany
  4.6.2. Gender-Specific Cases of Cystinuria in Germany
  4.6.3. Age-Specific Cases of Cystinuria in Germany
  4.6.4. Diagnosed Cases of Cystinuria in Germany
  4.6.5. Type-Specific Diagnosed Cases of Cystinuria in Germany
4.7. France
  4.7.1. Prevalent Cases of Cystinuria in France
  4.7.2. Gender-Specific Cases of Cystinuria in France
  4.7.3. Age-Specific Cases of Cystinuria in France
  4.7.4. Diagnosed Cases of Cystinuria in France
  4.7.5. Type-Specific Cases of Cystinuria in France
4.8. Italy
  4.8.1. Prevalent Cases of Cystinuria in Italy
  4.8.2. Gender-Specific Cases of Cystinuria in Italy
  4.8.3. Age-Specific Cases of Cystinuria in Italy
  4.8.4. Diagnosed Cases of Cystinuria in Italy
  4.8.5. Type-Specific Cases of Cystinuria in Italy
4.9. Spain
  4.9.1. Prevalent Cases of Cystinuria in Spain
  4.9.2. Gender-Specific Cases of Cystinuria in Spain
  4.9.3. Age-Specific Cases of Cystinuria in Spain
  4.9.4. Diagnosed Cases of Cystinuria in Spain
  4.9.5. Type-Specific Cases of Cystinuria in Spain
4.10. United Kingdom
  4.10.1. Prevalent Cases of Cystinuria in United Kingdom
  4.10.2. Gender-Specific Cases of Cystinuria in the United Kingdom
  4.10.3. Age-Specific Cases of Cystinuria in the United Kingdom
  4.10.4. Diagnosed Cases of Cystinuria in the United Kingdom
  4.10.5. Type-Specific Diagnosed Cases of Cystinuria in the United Kingdom
4.11. Japan
  4.11.1. Assumptions and Rationale
  4.11.2. Prevalent Cases of Cystinuria in Japan
  4.11.3. Gender-Specific Cases of Cystinuria in Japan
  4.11.4. Age-Specific Cases of Cystinuria in Japan
  4.11.5. Diagnosed Cases of Cystinuria in Japan
  4.11.6. Type-Specific Diagnosed Cases of Cystinuria in Japan

5. UNMET NEEDS

6. MARKET DRIVERS

7. MARKET BARRIERS

8. APPENDIX

8.1 Report Methodology

9. DELVEINSIGHT CAPABILITIES

10. DISCLAIMER

11. ABOUT DELVEINSIGHT

LIST OF TABLES

Table 1: Phenotypic classification of cystinuria based on parental (heterozygote) findings
Table 2: Urinary cystine concentration
Table 3: Genotypic classification of Cystinuria
Table 4: Most common Cystinuria-associated mutations in rBAT*, SLC3A1, b0,+AT‡, and SLC7A9
Table 5: Prevalent Patient Population of Cystinuria in 7MM (2017–2028)
Table 6: Prevalent Cases of Cystinuria in the United States (2017–2028)
Table 7: Gender-Specific Cases of Cystinuria in the United States (2017–2028)
Table 8: Age-Specific Cases of Cystinuria in the United States (2017–2028)
Table 9: Diagnosed Cases of Cystinuria in the United States (2017–2028)
Table 10: Type-Specific Cases of Cystinuria in the United States (2017–2028)
Table 11: Prevalent Cases of Cystinuria in Germany (2017–2028)
Table 12: Gender-Specific Cases of Cystinuria in Germany (2017–2028)
Table 13: Age-Specific Cases of Cystinuria in Germany (2017–2028)
Table 14: Diagnosed Cases of Cystinuria in Germany (2017–2028)
Table 15: Type-Specific Cases of Cystinuria in Germany (2017–2028)
Table 16: Prevalent Cases of Cystinuria in France (2017–2028)
Table 17: Gender-Specific Cases of Cystinuria in France (2017–2028)
Table 18: Age-Specific Cases of Cystinuria in France (2017–2028)
Table 19: Diagnosed Cases of Cystinuria in France (2017–2028)
Table 20: Type-Specific Cases of Cystinuria in France (2017–2028)
Table 21: Prevalent Cases of Cystinuria in Italy (2017–2028)
Table 22: Gender-Specific Cases of Cystinuria in Italy (2017–2028)
Table 23: Age-Specific Cases of Cystinuria in Italy (2017–2028)
Table 24: Diagnosed Cases of Cystinuria in Italy (2017–2028)
Table 25: Type-Specific Cases of Cystinuria in Italy (2017–2028)
Table 26: Prevalent Cases of Cystinuria in Spain (2017–2028)
Table 27: Gender-Specific Cases of Cystinuria in Spain (2017–2028)
Table 28: Age-Specific Cases of Cystinuria in Spain (2017–2028)
Table 29: Diagnosed Cases of Cystinuria in Spain (2017–2028)
Table 30: Type-Specific Cases of Cystinuria in Spain (2017–2028)
Table 31: Prevalent Cases of Cystinuria in the United Kingdom (2017–2028)
Table 32: Gender-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Table 33: Age-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Table 34: Diagnosed Cases of Cystinuria in the United Kingdom (2017–2028)
Table 35: Type-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Table 36: Prevalent Cases of Cystinuria in Japan (2017–2028)
Table 37: Gender-Specific Cases of Cystinuria in Japan (2017–2028)
Table 38: Age-Specific Cases of Cystinuria in Japan (2017–2028)
Table 39: Diagnosed Cases of Cystinuria in Japan (2017–2028)
Table 40: Type-Specific Cases of Cystinuria in Japan (2017–2028)

LIST OF FIGURES

Figure 1: Cellular localization and function of rBAT (SLC3A1) and b0,+AT (SLC7A9)
Figure 2: Genomic structure and putative function of the encoded protein regions of the Cystinuria genes
Figure 3: Trafficking of cystine and dibasic amino acids in epithelial cells of the renal proximal tubule or small intestine
Figure 4: Diagnostic algorithm of Cystinuria
Figure 5: Prevalent Patient Population of Cystinuria in 7MM (2017–2028)
Figure 6: Prevalent Cases of Cystinuria in the United States (2017–2028)
Figure 7: Gender-Specific Cases of Cystinuria in the United States (2017–2028)
Figure 8: Age-Specific Cases of Cystinuria in the United States (2017–2028)
Figure 9: Diagnosed Cases of Cystinuria in the United States (2017–2028)
Figure 10: Type-Specific Cases of Cystinuria in the United States (2017–2028)
Figure 11: Prevalent Cases of Cystinuria Germany (2017–2028)
Figure 12: Gender-Specific Cases of Cystinuria in Germany (2017–2028)
Figure 13: Age-Specific Cases of Cystinuria in Germany (2017–2028)
Figure 14: Diagnosed Cases of Cystinuria in Germany (2017–2028)
Figure 15: Type-Specific Cases of Cystinuria in Germany (2017–2028)
Figure 16: Prevalent Cases of Cystinuria in France (2017–2028)
Figure 17: Gender-Specific Cases of Cystinuria in France (2017–2028)
Figure 18: Age-Specific Cases of Cystinuria in France (2017–2028)
Figure 19: Diagnosed Cases of Cystinuria in France (2017–2028)
Figure 20: Type-Specific Cases of Cystinuria in France (2017–2028)
Figure 21: Prevalent Cases of Cystinuria in Italy (2017–2028)
Figure 22: Gender-Specific Cases of Cystinuria in Italy (2017–2028)
Figure 23: Age-Specific Cases of Cystinuria in Italy (2017–2028)
Figure 24: Diagnosed Cases of Cystinuria in Italy (2017–2028)
Figure 25: Type-Specific Cases of Cystinuria in Italy (2017–2028)
Figure 26: Prevalent Cases of Cystinuria in Spain (2017–2028)
Figure 27: Gender-Specific Cases of Cystinuria in Spain (2017–2028)
Figure 28: Age-Specific Cases of Cystinuria in Spain (2017–2028)
Figure 29: Diagnosed Cases of Cystinuria in Spain (2017–2028)
Figure 30: Type-Specific of Cystinuria in Spain (2017–2028)
Figure 31: Prevalent Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 32: Gender-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 33: Age-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 34: Diagnosed Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 35: Type-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 36: Prevalent Cases of Cystinuria in Japan (2017–2028)
Figure 37: Gender-Specific Cases of Cystinuria in Japan (2017–2028)
Figure 38: Age-Specific Cases of Cystinuria in Japan (2017–2028)
Figure 39: Diagnosed Cases of Cystinuria in Japan (2017–2028)
Figure 40: Type-Specific Cases of Cystinuria in Japan (2017–2028)
Figure 41: Unmet Needs
Figure 42: Market Drivers
Figure 43: Market Barriers


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