Epiomic Epidemiology Series: Lesch–nyhan Syndrome Forecast in 17 Major Markets 2017–2027

October 2017 | 42 pages | ID: E0BC01DD446EN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Series Forecast Report on Lesch-Nyhan Syndrome in 17 Major Markets

Lesch–Nyhan syndrome (LNS) is a rare X-linked recessive disease caused by mutations in the HPRT1 gene encoding the enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT). The disease manifests by severe hyperuricaemia resulting in motor dysfunction, intellectual disability and behavioural problems including recurrent self-injury. Many clinically relevant mutations in the HPRT1 gene have been found, suggesting that the level of residual enzyme activity is usually the key feature affecting the clinical image of the disease.

This report provides the current prevalent population for Lesch–Nyhan syndrome across 17 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Poland, Netherlands, Turkey, Japan, South Korea, India, Australia, Brazil, 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, some features of Lesch–Nyhan syndrome patients, as well as several of the main symptoms and co-morbidities 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 Lesch–Nyhan syndrome include:

• Various degree of physical disability
• Delayed growth and puberty
• Gouty arthritis with flare-ups
• Kidney and bladder stones
• Infections of self-inflicted wounds
• Megaloblastic anaemia
• Cardiovascular complications
• Pneumonia

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 Lesch–Nyhan syndrome market to target the development of future products, pricing strategies and launch plans.
• Gain further insight into the prevalence of the subdivided types of Lesch–Nyhan syndrome 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 Lesch–Nyhan syndrome’s prevalent population.
• Identify sub-populations within Lesch–Nyhan syndrome which require treatment.
• Gain an understanding of the specific markets that have the largest number of Lesch–Nyhan syndrome 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 LESCH–NYHAN SYNDROME

FEATURES OF LESCH–NYHAN SYNDROME PATIENTS

COMORBID CONDITIONS OF LESCH–NYHAN SYNDROME PATIENTS

  PATIENTS WIH SIGNS AND SYMPTOMS AT TIME OF DIAGNOSIS
  PATIENTS WIH SIGNS AND SYMPTOMS AFTER DIAGNOSIS

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. Prevalence of LNS, males (000s)
Table 2. Plasma uric acid levels (µmol/L) in LNS patients, males (000s)
Table 3. LNS patients with mother carrier, males (000s)
Table 4. LNS patients with signs/symptoms at time of diagnosis, males (000s)
Table 5. LNS patients with eating disorder at time of diagnosis, males (000s)
Table 6. LNS patients with self-injurious behaviour at time of diagnosis, males (000s)
Table 7. LNS patients with hypotonia at time of diagnosis, males (000s)
Table 8. LNS patients with developmental delay at time of diagnosis, males (000s)
Table 9. LNS patients with movement disorder at time of diagnosis, males (000s)
Table 10. LNS patients with cerebral palsy at time of diagnosis, males (000s)
Table 11. LNS patients with irritability at time of diagnosis, males (000s)
Table 12. LNS patients with renal failure at time of diagnosis, males (000s)
Table 13. LNS patients with crystals in diaper at time of diagnosis, males (000s)
Table 14. LNS patients with signs/symptoms after diagnosis, males (000s)
Table 15. LNS patients with eating problems after diagnosis, males (000s)
Table 16. LNS patients with self-injurious behaviour after diagnosis, males (000s)
Table 17. LNS patients with dystonia after diagnosis, males (000s)
Table 18. LNS patients with speech problem after diagnosis, males (000s)
Table 19. LNS patients with hiatus hernia after diagnosis, males (000s)
Table 20. LNS patients with gastrostomy after diagnosis, males (000s)
Table 21. LNS patients with teeth removed after diagnosis, males (000s)
Table 22. Abbreviations and Acronyms used in the report
Table 23. USA Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 24. Canada Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 25. France Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 26. Germany Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 27. Italy Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 28. Spain Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 29. UK Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 30. Poland Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 31. Netherlands Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 32. Turkey Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 33. Japan Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 34. South Korea Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 35. India Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 36. Australia Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 37. Brazil Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 38. Mexic  Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)
Table 39. Argentina Prevalence of Lesch–Nyhan syndrome by 5-yr age cohort, males (000s)