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.