DelveInsight has launched a new Report on “Nephropathic Cystinosis – Market Insights, Epidemiology, and Market Forecast-2030”.
DelveInsight’s “Nephropathic Cystinosis – Market Insights, Epidemiology, and Market Forecast-2030” report delivers an in-depth understanding of the Nephropathic Cystinosis , historical and forecasted epidemiology as well as the Nephropathic Cystinosis market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
Some of the Key Facts:
1. Cystinosis affects males and females in equal numbers. The disorder is estimated to occur in 1 in 100,000-200,000 people in the general population.
2. Cystinosis is the most common cause of renal Fanconi syndrome in children and accounts for approximately 5 percent of all childhood cases of kidney failure.
3. Nephropathic Cystinosis incidence is approximately 1:150,000 to 200,000 live births and its prevalence is approximately 1.6 per million population.
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Key benefits of the Report:
1. Nephropathic Cystinosis market report covers a descriptive overview and comprehensive insight of the Nephropathic Cystinosis epidemiology and Nephropathic Cystinosis market in the 7 MM (United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)
2. Nephropathic Cystinosis market report provides insights on the current and emerging therapies.
3. Nephropathic Cystinosis market report provides a global historical and forecasted market covering drug outreach in 7 MM.
4. Nephropathic Cystinosis market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Nephropathic Cystinosis market.
“Infantile (Nephropathic Cystinosis) is the most common form of the disease, accounting for 95 % of cases.”
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Nephropathic Cystinosis treatment is directed toward the specific symptoms that are apparent in each individual and is best treated by a team of specialists, who have knowledge about the disease, and who can offer supportive and symptomatic care. Care is directed toward relieving the symptoms of the disease. Cystine Depleting Therapies are now available for the Nephropathic Cystinosis treatment.
The ultimate goal of the Nephropathic Cystinosis treatment is to reduce the cystine levels in the body. The treatment approach involves cystine depleting therapies, symptomatic medications, and kidney transplant in severe cases. There are two cystine depleting therapies which are approved by the FDA for the treatment of this disease including Cystagon (Mylan Pharmaceuticals) and Procysbi (Horizon Pharma USA, Inc.). Cystagon (Mylan Pharmaceuticals) is approved for Nephropathic Cystinosis treatment in adults and children. It reacts with cystine to form another amino acid, called cysteine, and a compound called a cysteine-cysteamine salt. The body is able to remove this salt from the cells. The amount of cystine in the organs is therefore reduced, and this limits the damage to these organs.
PROCYSBI (Horizon Pharma USA, Inc.) is the other candidate which is approved for Nephropathic Cystinosis treatment in adult and pediatric patients 2 years of age and older. It participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.
Both of the therapies have received orphan drug designation from the USFDA. Apart from these therapies, certain symptomatic treatment is also advised and consequently happens to occupy a considerable share in the overall treatment landscape. Infants and young children with cystinosis receive fluid and electrolytes, such as sodium and potassium. Vitamin D and phosphate salts are also given to deal with the problems of weaker bones. Diabetes is usually managed with a low-sugar diet, insulin, or other medicines that help control sugar in the blood. In severe cases where none of the therapies is effective, kidney transplant is recommended.
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Key Companies Covered:
- Horizon Pharma/Raptor Pharmaceuticals
- Mylan Pharmaceuticals/Recordati
- Leadiant Biosciences
- Recordati S.p.A.
- Avrobio
- Eloxx Pharmaceuticals
- Mylan EPD
- And Many Others
Table of contents
1. Key Insights
2. Executive Summary of Nephropathic Cystinosis
3. Competitive Intelligence Analysis for Nephropathic Cystinosis
4. Nephropathic Cystinosis : Market Overview at a Glance
4.1. Nephropathic Cystinosis Total Market Share (%) Distribution in 2017
4.2. Nephropathic Cystinosis Total Market Share (%) Distribution in 2030
5. Nephropathic Cystinosis : Disease Background and Overview
5.1. Introduction
5.2. Sign and Symptoms
5.3. Pathophysiology
5.4. Risk Factors
5.5. Diagnosis
6. Patient Journey
7. Nephropathic Cystinosis Epidemiology and Patient Population
7.1. Epidemiology Key Findings
7.2. Assumptions and Rationale: 7MM
7.3. Epidemiology Scenario: 7MM
7.3.1. Nephropathic Cystinosis Epidemiology Scenario in the 7MM (2017-2030)
7.4. United States Epidemiology
7.4.1. Nephropathic Cystinosis Epidemiology Scenario in the United States (2017-2030)
7.5. EU-5 Country-wise Epidemiology
7.5.1. Germany Epidemiology
7.5.1.1. Nephropathic Cystinosis Epidemiology Scenario in Germany (2017-2030)
7.5.2. France Epidemiology
7.5.2.1. Nephropathic Cystinosis Epidemiology Scenario in France (2017-2030)
7.5.3. Italy Epidemiology
7.5.3.1. Nephropathic Cystinosis Epidemiology Scenario in Italy (2017-2030)
7.5.4. Spain Epidemiology
7.5.4.1. Nephropathic Cystinosis Epidemiology Scenario in Spain (2017-2030)
7.5.5. United Kingdom Epidemiology
7.5.5.1. Nephropathic Cystinosis Epidemiology Scenario in the United Kingdom (2017-2030)
7.5.6. Japan Epidemiology
7.5.6.1. Nephropathic Cystinosis Epidemiology Scenario in Japan (2017-2030)
8. Treatment Algorithm, Current Treatment, and Medical Practices
8.1. Nephropathic Cystinosis Treatment and Management
8.2. Nephropathic Cystinosis Treatment Algorithm
9. Unmet Needs
10. Key Endpoints of Nephropathic Cystinosis Treatment
11. Marketed Products
11.1. List of Marketed Products in the 7MM
11.2. Drug Name: Company Name
11.2.1. Product Description
11.2.2. Regulatory Milestones
11.2.3. Other Developmental Activities
11.2.4. Pivotal Clinical Trials
11.2.5. Summary of Pivotal Clinical Trial
List to be continued in report
12. Emerging Therapies
12.1. Key Cross
12.2. Drug Name: Company Name
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.4. Safety and Efficacy
12.2.5. Product Profile
List to be continued in report
13. Nephropathic Cystinosis : Seven Major Market Analysis
13.1. Key Findings
13.2. Nephropathic Cystinosis Market Size in 7MM
13.3. Nephropathic Cystinosis Market Size by Therapies in the 7MM
14. Attribute analysis
15. 7MM: Market Outlook
15.1. United States: Market Size
15.1.1. Nephropathic Cystinosis Total Market Size in the United States
15.1.2. Nephropathic Cystinosis Market Size by Therapies in the United States
15.2. EU-5 countries: Market Size and Outlook
15.3. Germany Market Size
15.3.1. Nephropathic Cystinosis Total Market Size in Germany
15.3.2. Nephropathic Cystinosis Market Size by Therapies in Germany
15.4. France Market Size
15.4.1. Nephropathic Cystinosis Total Market Size in France
15.4.2. Nephropathic Cystinosis Market Size by Therapies in France
15.5. Italy Market Size
15.5.1. Nephropathic Cystinosis Total Market Size in Italy
15.5.2. Nephropathic Cystinosis Market Size by Therapies in Italy
15.6. Spain Market Size
15.6.1. Nephropathic Cystinosis Total Market Size in Spain
15.6.2. Nephropathic Cystinosis Market Size by Therapies in Spain
15.7. United Kingdom Market Size
15.7.1. Nephropathic Cystinosis Total Market Size in the United Kingdom
15.7.2. Nephropathic Cystinosis Market Size by Therapies in the United Kingdom
15.8. Japan Market Outlook
15.8.1. Japan Market Size
15.8.2. Nephropathic Cystinosis Total Market Size in Japan
15.8.3. Nephropathic Cystinosis Market Size by Therapies in Japan
16. Access and Reimbursement Overview of Nephropathic Cystinosis
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
20.1. Bibliography
20.2. Report Methodology
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
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