Epiomic Epidemiology Series: Cerebrotendinous Xanthomatosis Forecast in 18 Major Markets 2017-2027

September 2017 | 54 pages | ID: E3B752E6C1EEN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Series Forecast Report on Cerebrotendinous Xanthomatosis in 18 Major Markets

Cerebrotendinous xanthomatosis (CTX) is a rare autosomal recessive lipid storage disease caused by mutations in the CYP27A1 gene encoding a cytochrome P450 oxidase, also known as sterol 27-hydroxylase. The lack of the functional enzyme results in a reduced production of chenodeoxycholic acid (CDCA) and cholic acid (CA), components of bile, as well as accumulation of cholestanol and cholesterol in different tissues, e.g., brain, tendons, lenses, bones and vessels. No genotype–phenotype correlations have been identified, and the phenotypic manifestation can vary even between identical twins.

This report provides the current prevalent population for CTX across 18 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Japan, Russia, Brazil, Poland, Austria, The Netherlands, Turkey, India, China, Mexico, Argentina) split by gender and 5-year age cohort. Along with the current prevalence, the report also contains a disease overview of the risk factors, disease diagnosis and prognosis along with specific variations by geography and ethnicity.

Providing a value-added level of insight from the analysis team at Black Swan, several of the main symptoms and co-morbidities of CTX have been quantified and presented alongside the overall prevalence figures. These sub-populations within the main disease are also included at a country level across the 10-year forecast snapshot.

Main symptoms and co-morbidities for CTX include:

• Chronic diarrhoea
• Developmental cataracts
• Tendon xanthomas
• Seizures
• Intellectual impairment
• Osteoporosis

This report is built using data and information sourced from the proprietary Epiomic patient segmentation database. To generate accurate patient population estimates, the Epiomic database utilises a combination of several world class sources that deliver the most up to date information form patient registries, clinical trials and epidemiology studies. All of the sources used to generate the data and analysis have been identified in the report.

Reason to buy

• Able to quantify patient populations in global CTX market to target the development of future products, pricing strategies and launch plans.
• Gain further insight into the prevalence of the subdivided types of CTX and identify patient segments with high potential.
• Delivery of more accurate information for clinical trials in study sizing and realistic patient recruitment for various countries.
• Provide a level of understanding on the impact from specific co-morbid conditions on CTX prevalent population.
• Identify sub-populations within CTX which require treatment.
• Gain an understanding of the specific markets that have the largest number of CTX patients.

Contents

INTRODUCTION

CAUSE OF THE DISEASE

RISK FACTORS & PREVENTION

DIAGNOSIS OF THE DISEASE

VARIATION BY GEOGRAPHY/ETHNICITY

DISEASE PROGNOSIS & CLINICAL COURSE

KEY COMORBID CONDITIONS / FEATURES ASSOCIATED WITH THE DISEASE

METHODOLOGY FOR QUANTIFICATION OF PATIENT NUMBERS

TOP-LINE PREVALENCE FOR CEREBROTENDINOUS XANTHOMATOSIS

COMORBID CONDITIONS OF CEREBROTENDINOUS XANTHOMATOSIS PATIENTS

ABBREVIATIONS USED IN THE REPORT

OTHER BLACK SWAN ANALYSIS PUBLICATIONS

BLACK SWAN ANALYSIS ONLINE PATIENT-BASED DATABASES

PATIENT-BASED OFFERING

ONLINE PRICING DATA & PLATFORMS

REFERENCES

APPENDIX

List Of Tables

LIST OF TABLES AND FIGURES

Table 1. The varied manifestations of cerebrotendinous xanthomatosis
Table 2. Prevalence of Cerebrotendinous Xanthomatosis, total (000s)
Table 3. Prevalence of Cerebrotendinous Xanthomatosis, males (000s)
Table 4. Prevalence of Cerebrotendinous Xanthomatosis, females (000s)
Table 5. CTX Patients with Cataracts, total (000s)
Table 6. CTX Patients with Intellectual Impairment, total (000s)
Table 7. CTX Patients with Seizures, total (000s)
Table 8. CTX Patients with Tendon Xanthomas, total (000s)
Table 9. CTX Patients with Osteoporosis, total (000s)
Table 10. CTX Patients with Diarrhoea, total (000s)
Table 11. Abbreviations and Acronyms used in the report
Table 12. USA Prevalence of CTX by 5-yr age cohort, males (000s)
Table 13. USA Prevalence of CTX by 5-yr age cohort, females (000s)
Table 14. Canada Prevalence of CTX by 5-yr age cohort, males (000s)
Table 15. Canada Prevalence of CTX by 5-yr age cohort, females (000s)
Table 16. France Prevalence of CTX by 5-yr age cohort, males (000s)
Table 17. France Prevalence of CTX by 5-yr age cohort, females (000s)
Table 18. Germany Prevalence of CTX by 5-yr age cohort, males (000s)
Table 19. Germany Prevalence of CTX by 5-yr age cohort, females (000s)
Table 20. Italy Prevalence of CTX by 5-yr age cohort, males (000s)
Table 21. Italy Prevalence of CTX by 5-yr age cohort, females (000s)
Table 22. Spain Prevalence of CTX by 5-yr age cohort, males (000s)
Table 23. Spain Prevalence of CTX by 5-yr age cohort, females (000s)
Table 24. UK Prevalence of CTX by 5-yr age cohort, males (000s)
Table 25. UK Prevalence of CTX by 5-yr age cohort, females (000s)
Table 26. Japan Prevalence of CTX by 5-yr age cohort, males (000s)
Table 27. Japan Prevalence of CTX by 5-yr age cohort, females (000s)
Table 28. Russia Prevalence of CTX by 5-yr age cohort, males (000s)
Table 29. Russia Prevalence of CTX by 5-yr age cohort, females (000s)
Table 30. Brazil Prevalence of CTX by 5-yr age cohort, males (000s)
Table 31. Brazil Prevalence of CTX by 5-yr age cohort, females (000s)
Table 32. Poland Prevalence of CTX by 5-yr age cohort, males (000s)
Table 33. Poland Prevalence of CTX by 5-yr age cohort, females (000s)
Table 34. Austria Prevalence of CTX by 5-yr age cohort, males (000s)
Table 35. Austria Prevalence of CTX by 5-yr age cohort, females (000s)
Table 36. The Netherlands Prevalence of CTX by 5-yr age cohort, males (000s)
Table 37. The Netherlands Prevalence of CTX by 5-yr age cohort, females (000s)
Table 38. Turkey Prevalence of CTX by 5-yr age cohort, males (000s)
Table 39. Turkey Prevalence of CTX by 5-yr age cohort, females (000s)
Table 40. India Prevalence of CTX by 5-yr age cohort, males (000s)
Table 41. India Prevalence of CTX by 5-yr age cohort, females (000s)
Table 42. China Prevalence of CTX by 5-yr age cohort, males (000s)
Table 43. China Prevalence of CTX by 5-yr age cohort, females (000s)
Table 44. Mexico Prevalence of CTX by 5-yr age cohort, males (000s)
Table 45. Mexico Prevalence of CTX by 5-yr age cohort, females (000s)
Table 46. Argentina Prevalence of CTX by 5-yr age cohort, males (000s)
Table 47. Argentina Prevalence of CTX by 5-yr age cohort, females (000s)