Cystinosis Epidemiology Forecast

DelveInsight’s ‘Cystinosis—Epidemiology Forecast–2030’ report delivers an in-depth understanding of the Cystinosis, historical and forecasted epidemiology as well as the Cystinosis trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Geographies Covered

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

Study Period: 2018–2030

Cystinosis Disease Understanding

Cystinosis is a rare autosomal recessive metabolic disorder in which cystinosin gene (CTNS) mutations result in defective cystine transport across the lysosomal membrane and accumulation and crystallization (disulfide-linked dimers of the amino acid cysteine) in all tissues with progressive and widespread functional impairment of kidneys, thyroid, testis, pancreas, muscle, brain, and eye. In the eye, cystine crystal formation and accumulation in the cornea leads to photophobia, blepharospasm, corneal erosions, superficial punctate keratopathy, and band keratopathy. Without treatment, this can result in a complete loss of vision requiring keratoplasty.

 

Cystinosis is a condition in which the body accumulates the amino acid cystine (a building block of proteins) within cells. Excess cystine forms crystals that can build up and damage cells. These crystals negatively affect many systems in the body, especially the kidneys and eyes. There are three distinct types of cystinosis. In order of decreasing severity, they are nephropathic cystinosis (Infantile Nephropathic), intermediate cystinosis (Late Onset or juvenile cystinosis), and non-nephropathic or ocular cystinosis.

Continued in the report…..

Cystinosis Epidemiology Perspective by DelveInsight

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalence of Cystinosis, Diagnosed Prevalence of Cystinosis and Type-specific Diagnosed Prevalence of Cystinosis scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2018 to 2030.

Cystinosis Detailed Epidemiology Segmentation

  • Total prevalent cases of cystinosis in the 7MM were found to be 3,008 in 2020, which might increase in 2030. These prevalent cases are expected to increase during the study period (2018-2030).
  • The total diagnosed prevalent cases of cystinosis in 7MM are anticipated to reach 1,472 in 2030.
  • Among 7MM, the United States has the highest diagnosed prevalent cases of cystinosis with 655 cases in 2020. In EU-5, the UK has the highest number of cases, i.e., 222, followed by Germany with 183 cases. While Italy has the least number of cases with 43 in 2020.
  • In 2020, there was a total of 599 cases of Infantile nephropathic cystinosis, 33 cases of Juvenile nephropathic cystinosis, and 23 cases of Adult-onset (Ocular, or non-nephropathic cystinosis) in the US. These cases are expected to increase by 2030.
  • Japan had 23 diagnosed prevalent cases of cystinosis in 2020. These cases are expected to decrease. Out of the total diagnosed cases, 21 cases account for infantile nephropathic cystinosis, 1 case of Juvenile nephropathic cystinosis, and 1 case of Adult-onset in 2020.

Scope of the Report

  • The report covers the descriptive overview of Cystinosis, explaining its causes, symptoms, pathophysiology, and genetic basis.
  • The report provides insight into the 7MM historical and forecasted patient pool covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
  • The report assesses the disease risk and burden and highlights the unmet needs of Cystinosis.
  • The report helps to recognize the growth opportunities in the 7MM concerning the patient population.
  • The report provides the segmentation of the disease epidemiology for 7MM by Prevalence of Cystinosis, Diagnosed Prevalence of Cystinosis, and Type-specific Diagnosed Prevalence of Cystinosis.

Report Highlights

  • 10-Year Forecast of Cystinosis
  • 7MM Coverage
  • Prevalence of Cystinosis
  • Diagnosed Prevalence of Cystinosis
  • Type-specific Diagnosed Prevalence of Cystinosis

Key Questions Answered

  • What are the disease risk, and burdens of Cystinosis?
  • What is the historical Cystinosis patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK), and Japan?
  • What would be the forecasted patient pool of Cystinosis at the 7MM level?
  • What will be the growth opportunities across the 7MM for the patient population about Cystinosis?
  • Out of the above-mentioned countries, which country would have the highest prevalent population of Cystinosis during the forecast period (2021–2030)?
  • At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?

Reasons to buy

The Cystinosis report will allow the user to -

  • Develop business strategies by understanding the trends shaping and driving the 7MM Cystinosis epidemiology forecast.
  • The Cystinosis epidemiology report and model were written and developed by Masters and Ph.D. level epidemiologists.
  • The Cystinosis 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 the 10-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

1. Key Insights

2. Report Introduction

3. Executive Summary of Cystinosis

4. Key Events

5. Disease Background and Overview

5.1. Introduction

5.2. Classification of Cystinosis

5.2.1. Nephropathic cystinosis

5.2.1.1. Symptoms and Associated Complications

5.2.2. Intermediate cystinosis/Late-Onset/Juvenile cystinosis

5.2.2.1. Symptoms

5.2.3. Non-nephropathic or ocular cystinosis

5.3. Cause of Cystinosis

5.4. Pathophysiology of Cystinosis

5.4.1. Cellular events in cystinosis related to cystine accumulation

5.4.2. Cellular events in cystinosis likely unrelated to cystine accumulation

5.5. Clinical description and complications

5.6. Genetic Basis of Cystinosis

5.6.1. Functional Characteristics of CTNS Mutations: Genotype-Phenotype Correlations

5.7. Diagnostics and Biomarker Follow-up

5.7.1. Differential Diagnosis and Biomarker Follow-up

5.7.2. Diagnostic Guidelines

5.7.2.1. The European Reference Network for Rare Kidney Diseases

6. Treatment and Management of Cystinosis

6.1. Symptomatic treatment

6.2. Cystine-depleting therapy

6.3. Therapeutic monitoring and Prognosis

6.4. Treatment Guidelines for Cystinosis

6.4.1. The European Reference Network for Rare Kidney Diseases

7. Epidemiology and Patient Population

7.1. Key Findings

7.2. Methodology

7.3. Total Prevalent Cases of Cystinosis in the 7MM

7.4. Diagnosed Prevalent Cases of Cystinosis in the 7MM

7.5. The United States

7.5.1. Assumptions and Rationale

7.5.2. Total Prevalent Cases of Cystinosis in the United States

7.5.3. Diagnosed Prevalent Cases of Cystinosis in the United States

7.5.4. Type-specific Diagnosed Prevalent Cases of Cystinosis in the United States

7.6. EU5

7.6.1. Total Prevalent Cases of Cystinosis in EU-5

7.6.2. Diagnosed Prevalent Cases of Cystinosis in EU-5

7.6.3. Type-specific Diagnosed Prevalent Cases of Cystinosis in EU-5

7.7. Japan

7.7.1. Assumptions and Rationale

7.7.2. Total Prevalent Cases of Cystinosis in Japan

7.7.3. Diagnosed Prevalent Cases of Cystinosis in Japan

7.7.4. Type-specific Diagnosed Prevalent Cases of Cystinosis in Japan

8. Appendix

8.1. Report Methodology

8.2. Bibliography

9. DelveInsight Capabilities

10. Disclaimer

11. About DelveInsight

List of Table

Table 1: Summary of Epidemiology (2018-2030)

Table 2: Key Events

Table 3: CTNS mutations and their corresponding phenotype, cystine transport activity, and location

Table 4: Total Prevalent Cases of Cystinosis in the 7MM (2018-2030)

Table 5: Diagnosed Prevalent Cases of Cystinosis in the 7MM (2018-2030)

Table 6: Total Prevalent cases of Cystinosis in the United States (2018-2030)

Table 7: Diagnosed Prevalent cases of Cystinosis in the United States (2018-2030)

Table 8: Type-specific Diagnosed Prevalent Cases of Cystinosis in the United States (2018-2030)

Table 9: Total Prevalent Cases of Cystinosis in EU-5 (2018-2030)

Table 10: Diagnosed Prevalent Cases of Cystinosis in EU-5 (2018-2030)

Table 11: Type-specific Diagnosed Prevalent Cases of Cystinosis in EU5 (2018-2030)

Table 12: Total Prevalent cases of Cystinosis in Japan (2018-2030)

Table 13: Diagnosed Prevalent cases of Cystinosis in Japan (2018-2030)

Table 14: Type-specific Diagnosed Prevalent Cases of cystinosis in Japan (2018-2030)

List of Figures

Figure 1: Structure of Human Immunodeficiency Virus (HIV)

Figure 2: Classification of Cystinosis

Figure 3: Symptoms of different types of Cystinosis

Figure 4: Schematic representation of the CTNS gene and all reported mutations in cystinosis

Figure 5: Simplified scheme of the current knowledge on the pathogenesis of cystinosis

Figure 6: Pathophysiology of cystinosis

Figure 7: Structure of cystinosin with the indication of CTNS mutations having an effect on LAMP2A localization, interaction with V-ATPase-Regulator-Rag complex and glycosylation status

Figure 8: Total Prevalent Cases of Cystinosis in the 7MM (2018-2030)

Figure 9: Diagnosed Prevalent Cases of Cystinosis in the 7MM (2018-2030)

Figure 10: Total Prevalent cases of Cystinosis in the United States (2018-2030)

Figure 11: Diagnosed Prevalent cases of Cystinosis in the United States (2018-2030)

Figure 12: Type-specific Diagnosed Prevalent Cases of Cystinosis in the United States (2018-2030)

Figure 13: Total Prevalent Cases of Cystinosis in EU-5 (2018-2030)

Figure 14: Diagnosed Prevalent Cases of Cystinosis in EU-5 (2018-2030)

Figure 15: Type-specific Diagnosed Prevalent cases of Cystinosis in EU-5

Figure 16: Total Prevalent cases of Cystinosis in Japan (2018-2030)

Figure 17: Diagnosed Prevalent cases of Cystinosis in Japan (2018-2030)

  • Tags:
  • Cystinosis Epidemiology
  • Cystinosis
  • Cystinosis Pipeline
  • Cystinosis Companies
  • Cystinosis prevalent population
  • Cystinosis incident population
  • Cystinosis patients diagnosed
  • Cystinosis treatment algorithm

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