Epiomic Epidemiology Series: Tay–Sachs Disease Forecast in 19 Major Markets 2018–2028

June 2018 | 78 pages | ID: E94F48325F2EN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Series Forecast Report on Tay Sachs Disease (TSD) in 19 Major Markets

Hexosaminidase A deficiency (HEX A deficiency) results in a group of neurodegenerative disorders caused by the dysfunctional activity of the specific glycosphingolipid GM2 ganglioside. The disease occurs when cell membrane components known as gangliosides, accumulate to toxic levels, particularly in neurons in the brain and spinal cord, leading to the destruction of neurons and interference with biological functions. The predominant HEX A deficiency is Tay-Sachs disease (TSD) with its common acute infantile variant, which begins around six months of age and usually results in death by the age of four. Alternative variants of the pathology are the juvenile and the adult-onset TSD types.

This report provides the current prevalent population for TSD disease across 19 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Poland, Netherlands, Czech Republic, Portugal, Russia, Turkey, Saudi Arabia, Japan, China, India, Australia, Brazil) 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, patients with main phenotypes and multiple symptoms of TSD 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 TSD include:

• Visual problems
• Low muscle tone
• Seizures
• Neuromuscular abnormalities
• Speech disturbances
• Cerebellar dysfunctions/Impaired cognitive status

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 from 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 TSD market to target the development of future products, pricing strategies and launch plans.
• Gain further insight into the prevalence of the subdivided types of TSD 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 TSD prevalent population.
• Identify sub-populations within TSD which require treatment.
• Gain an understanding of the specific markets that have the largest number of TSD 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 TAY–SACHS DISEASE
FEATURES OF TAY–SACHS DISEASE PATIENTS
INFANTILE PHENOTYPE
JUVENILE PHENOTYPE
ADULT (LOTS) PHENOTYPE
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. Prevalence of TSD, total (000s)
Table 2. Prevalence of TSD, males (000s)
Table 3. Prevalence of TSD, females (000s)
Table 4. Patients with TSD by phenotype, total (000s)
Table 5. Infantile-phenotype TSD patients with hypertonia, total (000s)
Table 6. Infantile-phenotype TSD patients with myotatic reflexes, total (000s)
Table 7. Patients with infantile-phenotype TSD by status of myotatic reflexes, total (000s)
Table 8. Infantile-phenotype TSD patients with cherry-red spot, total (000s)
Table 9. Infantile-phenotype TSD patients with seizures, total (000s)
Table 10. Juvenile-phenotype TSD patients with gait disturbances, total (000s)
Table 11. Juvenile-phenotype TSD patients with speech disturbances, total (000s)
Table 12. Juvenile-phenotype TSD patients with intellectual delay/deficit, total (000s)
Table 13. Juvenile-phenotype TSD patients with muscle wasting, total (000s)
Table 14. Juvenile-phenotype TSD patients with urinary/faecal incontinence, total (000s)
Table 15. Juvenile-phenotype TSD patients with seizures, total (000s)
Table 16. Juvenile-phenotype TSD patients with dysphagia, total (000s)
Table 17. Juvenile-phenotype TSD patients with behavioural/psychiatric disturbances, total (000s)
Table 18. Juvenile-phenotype TSD patients with vision problems, total (000s)
Table 19. Adult (LOTS)-phenotype TSD patients with speech disturbances, total (000s)
Table 20. Adult (LOTS)-phenotype TSD patients with stuttering, total (000s)
Table 21. Adult (LOTS)-phenotype TSD patients with dysarthria, total (000s)
Table 22. Adult (LOTS)-phenotype TSD patients with ocular findings, total (000s)
Table 23. Adult (LOTS)-phenotype TSD patients with saccade abnormality, total (000s)
Table 24. Adult (LOTS)-phenotype TSD patients with gaze palsy, total (000s)
Table 25. Adult (LOTS)-phenotype TSD patients with movement disorders, total (000s)
Table 26. Adult (LOTS)-phenotype TSD patients with tremor, total (000s)
Table 27. Adult (LOTS)-phenotype TSD patients with dystonia, total (000s)
Table 28. Adult (LOTS)-phenotype TSD patients with dyskinesia, total (000s)
Table 29. Adult (LOTS)-phenotype TSD patients with neuromuscular abnormalities, total (000s)
Table 30. Adult (LOTS)-phenotype TSD patients with weakness/wasting, total (000s)
Table 31. Adult (LOTS)-phenotype TSD patients with fasciculations, total (000s)
Table 32. Adult (LOTS)-phenotype TSD patients with cramps, total (000s)
Table 33. Adult (LOTS)-phenotype TSD patients with reflex changes, total (000s)
Table 34. Adult (LOTS)-phenotype TSD patients with hypotonia, total (000s)
Table 35. Adult (LOTS)-phenotype TSD patients with hypertonia, total (000s)
Table 36. Adult (LOTS)-phenotype TSD patients with Babinski sign, total (000s)
Table 37. Adult (LOTS)-phenotype TSD patients with primitive reflexes, total (000s)
Table 38. Adult (LOTS)-phenotype TSD patients with gait disturbances, total (000s)
Table 39. Adult (LOTS)-phenotype TSD patients with ataxia, total (000s)
Table 40. Adult (LOTS)-phenotype TSD patients with wide-based gait disturbances, total (000s)
Table 41. Adult (LOTS)-phenotype TSD patients with inability to ambulate, total (000s)
Table 42. Adult (LOTS)-phenotype TSD patients with cerebellar dysfunction, total (000s)
Table 43. Adult (LOTS)-phenotype TSD patients with sensory loss, total (000s)
Table 44. Abbreviations and acronyms used in the report
Table 45. USA prevalence of TSD by 5-yr age cohort, males (000s)
Table 46. USA prevalence of TSD by 5-yr age cohort, females (000s)
Table 47. Canada prevalence of TSD by 5-yr age cohort, males (000s)
Table 48. Canada prevalence of TSD by 5-yr age cohort, females (000s)
Table 49. France prevalence of TSD by 5-yr age cohort, males (000s)
Table 50. France prevalence of TSD by 5-yr age cohort, females (000s)
Table 51. Germany prevalence of TSD by 5-yr age cohort, males (000s)
Table 52. Germany prevalence of TSD by 5-yr age cohort, females (000s)
Table 53. Italy prevalence of TSD by 5-yr age cohort, males (000s)
Table 54. Italy prevalence of TSD by 5-yr age cohort, females (000s)
Table 55. Spain prevalence of TSD by 5-yr age cohort, males (000s)
Table 56. Spain prevalence of TSD by 5-yr age cohort, females (000s)
Table 57. UK prevalence of TSD by 5-yr age cohort, males (000s)
Table 58. UK prevalence of TSD by 5-yr age cohort, females (000s)
Table 59. Poland prevalence of TSD by 5-yr age cohort, males (000s)
Table 60. Poland prevalence of TSD by 5-yr age cohort, females (000s)
Table 61. Netherlands prevalence of TSD by 5-yr age cohort, males (000s)
Table 62. Netherlands prevalence of TSD by 5-yr age cohort, females (000s)
Table 63. Czech Republic prevalence of TSD by 5-yr age cohort, males (000s)
Table 64. Czech Republic prevalence of TSD by 5-yr age cohort, females (000s)
Table 65. Portugal prevalence of TSD by 5-yr age cohort, males (000s)
Table 66. Portugal prevalence of TSD by 5-yr age cohort, females (000s)
Table 67. Russia prevalence of TSD by 5-yr age cohort, males (000s)
Table 68. Russia prevalence of TSD by 5-yr age cohort, females (000s)
Table 69. Turkey prevalence of TSD by 5-yr age cohort, males (000s)
Table 70. Turkey prevalence of TSD by 5-yr age cohort, females (000s)
Table 71. Saudi Arabia prevalence of TSD by 5-yr age cohort, males (000s)
Table 72. Saudi Arabia prevalence of TSD by 5-yr age cohort, females (000s)
Table 73. Japan prevalence of TSD by 5-yr age cohort, males (000s)
Table 74. Japan prevalence of TSD by 5-yr age cohort, females (000s)
Table 75. China prevalence of TSD by 5-yr age cohort, males (000s)
Table 76. China prevalence of TSD by 5-yr age cohort, females (000s)
Table 77. India prevalence of TSD by 5-yr age cohort, males (000s)
Table 78. India prevalence of TSD by 5-yr age cohort, females (000s)
Table 79. Australia prevalence of TSD by 5-yr age cohort, males (000s)
Table 80. Australia prevalence of TSD by 5-yr age cohort, females (000s)
Table 81. Brazil prevalence of TSD by 5-yr age cohort, males (000s)
Table 82. Brazil prevalence of TSD by 5-yr age cohort, females (000s)