Epiomic Epidemiology Series: Mucopolysaccharidosis Type I Forecast in 27 Major Markets 2018–2028

April 2018 | 74 pages | ID: EA33DE74135EN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Forecast Report on Mucopolysaccharidosis Type I in 27 Major Markets

Mucopolysaccharidosis type I (MPS I) is a recessively inherited autosomal genetic disorder in which glycosaminoglycans (GAGs) are accumulated in lysosomes due to mutations that cause loss of function of an enzyme responsible for degrading GAGs. The disorder can occur in one of three forms, all of which involve tissue damage and multisystem signs and symptoms but differ in severity and rate of progression, with the most severe form characterised by pre-teen mortality of patients. The disorder constitutes a serious burden not only to the patients, but also to their families, and its management requires a multidisciplinary approach aimed to improve both the physical and the neuropsychological manifestations.

This report provides the current prevalent population for MPS I across 27 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Poland, Netherlands, Norway, Sweden, Denmark, Switzerland, Ireland, Portugal, Czech Republic, Russia, Turkey, Saudi Arabia, Japan, China, South Korea, India, Australia, Brazil, Mexico, Argentina) split by gender and 5-year age cohort. In addition to the current prevalence, the report provides an overview of the risk factors, diagnosis and prognosis of the disease, along with specific variations by geography and ethnicity.

Providing a value-added level of insight from the analysis team at Black Swan, MPS I patients grouped by phenotypes 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 of MPS I include:

• Inguinal and umbilical hernias
• Joint mobility problems (such as carpal tunnel syndrome)
• Chronic nasal discharge
• Recurrent upper respiratory tract infections
• Obstructive lung disease
• Ear infections
• Corneal clouding and retinal degeneration
• Gastrointestinal problems
• Hirsutism
• Sleep problems

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

Ability to quantify patient populations in global MPS I market to target the development of future products, pricing strategies and launch plans.

Further insight into the prevalence of the subdivided types of MPS I and identification of patient segments with high potential.

Delivery of more accurate information for clinical trials in study sizing and realistic patient recruitment for various countries.

Identification of MPS I patient sub-populations that require treatment.

Better understanding of the specific markets that have the largest number of MPS I 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 MUCOPOLYSACCHARIDOSIS TYPE I

FEATURES OF MUCOPOLYSACCHARIDOSIS TYPE I PATIENTS

ABBREVIATIONS USED IN THE REPORT

OTHER BLACK SWAN SERVICES & SOLUTIONS

REPORTS & PUBLICATIONS

ONLINE EPIDEMIOLOGY DATABASES

ONLINE PHARMACEUTICAL PRICING DATABASE

REFERENCES

APPENDIX

List Of Tables

LIST OF TABLES AND FIGURES

Table 1. Three major clinical subgroups of MPS I
Table 2. Prevalence of MPS I, total (000s)
Table 3. Prevalence of MPS I, males (000s)
Table 4. Prevalence of MPS I, females (000s)
Table 5. Patients with MPS I by phenotype, total (000s)
Table 6. Abbreviations and acronyms used in the report
Table 7. USA prevalence of MPS I by 5-yr age cohort, males (000s)
Table 8. USA prevalence of MPS I by 5-yr age cohort, females (000s)
Table 9. Canada prevalence of MPS I by 5-yr age cohort, males (000s)
Table 10. Canada prevalence of MPS I by 5-yr age cohort, females (000s)
Table 11. France prevalence of MPS I by 5-yr age cohort, males (000s)
Table 12. France prevalence of MPS I by 5-yr age cohort, females (000s)
Table 13. Germany prevalence of MPS I by 5-yr age cohort, males (000s)
Table 14. Germany prevalence of MPS I by 5-yr age cohort, females (000s)
Table 15. Italy prevalence of MPS I by 5-yr age cohort, males (000s)
Table 16. Italy prevalence of MPS I by 5-yr age cohort, females (000s)
Table 17. Spain prevalence of MPS I by 5-yr age cohort, males (000s)
Table 18. Spain prevalence of MPS I by 5-yr age cohort, females (000s)
Table 19. UK prevalence of MPS I by 5-yr age cohort, males (000s)
Table 20. UK prevalence of MPS I by 5-yr age cohort, females (000s)
Table 21. Poland prevalence of MPS I by 5-yr age cohort, males (000s)
Table 22. Poland prevalence of MPS I by 5-yr age cohort, females (000s)
Table 23. Netherlands prevalence of MPS I by 5-yr age cohort, males (000s)
Table 24. Netherlands prevalence of MPS I by 5-yr age cohort, females (000s)
Table 25. Norway prevalence of MPS I by 5-yr age cohort, males (000s)
Table 26. Norway prevalence of MPS I by 5-yr age cohort, females (000s)
Table 27. Sweden prevalence of MPS I by 5-yr age cohort, males (000s)
Table 28. Sweden prevalence of MPS I by 5-yr age cohort, females (000s)
Table 29. Denmark prevalence of MPS I by 5-yr age cohort, males (000s)
Table 30. Denmark prevalence of MPS I by 5-yr age cohort, females (000s)
Table 31. Switzerland prevalence of MPS I by 5-yr age cohort, males (000s)
Table 32. Switzerland prevalence of MPS I by 5-yr age cohort, females (000s)
Table 33. Ireland prevalence of MPS I by 5-yr age cohort, males (000s)
Table 34. Ireland prevalence of MPS I by 5-yr age cohort, females (000s)
Table 35. Portugal prevalence of MPS I by 5-yr age cohort, males (000s)
Table 36. Portugal prevalence of MPS I by 5-yr age cohort, females (000s)
Table 37. Czech Republic prevalence of MPS I by 5-yr age cohort, males (000s)
Table 38. Czech Republic prevalence of MPS I by 5-yr age cohort, females (000s)
Table 39. Russia prevalence of MPS I by 5-yr age cohort, males (000s)
Table 40. Russia prevalence of MPS I by 5-yr age cohort, females (000s)
Table 41. Turkey prevalence of MPS I by 5-yr age cohort, males (000s)
Table 42. Turkey prevalence of MPS I by 5-yr age cohort, females (000s)
Table 43. Saudi Arabia prevalence of MPS I by 5-yr age cohort, males (000s)
Table 44. Saudi Arabia prevalence of MPS I by 5-yr age cohort, females (000s)
Table 45. Japan prevalence of MPS I by 5-yr age cohort, males (000s)
Table 46. Japan prevalence of MPS I by 5-yr age cohort, females (000s)
Table 47. China prevalence of MPS I by 5-yr age cohort, males (000s)
Table 48. China prevalence of MPS I by 5-yr age cohort, females (000s)
Table 49. South Korea prevalence of MPS I by 5-yr age cohort, males (000s)
Table 50. South Korea prevalence of MPS I by 5-yr age cohort, females (000s)
Table 51. India prevalence of MPS I by 5-yr age cohort, males (000s)
Table 52. India prevalence of MPS I by 5-yr age cohort, females (000s)
Table 53. Australia prevalence of MPS I by 5-yr age cohort, males (000s)
Table 54. Australia prevalence of MPS I by 5-yr age cohort, females (000s)
Table 55. Brazil prevalence of MPS I by 5-yr age cohort, males (000s)
Table 56. Brazil prevalence of MPS I by 5-yr age cohort, females (000s)
Table 57. Mexico prevalence of MPS I by 5-yr age cohort, males (000s)
Table 58. Mexico prevalence of MPS I by 5-yr age cohort, females (000s)
Table 59. Argentina prevalence of MPS I by 5-yr age cohort, males (000s)
Table 60. Argentina prevalence of MPS I by 5-yr age cohort, females (000s)