EPIOMIC EPIDEMIOLOGY SERIES: MUCOPOLYSACCHARIDOSIS TYPE IV FORECAST IN 28 MAJOR MARKETS 2018–2028

Black Swan Analysis Epiomic™ Epidemiology Forecast Report on Mucopolysaccharidosis Type IV in 28 Major Markets

Mucopolysaccharidosis type IV (MPS IV), also known as Morquio syndrome, is an inherited autosomal recessive 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 manifests with multiple skeletal defects and has two subtypes, MPS IVA and MPS IVB, differing in the mutated gene. MPS IV constitutes a burden to the patients and their families as the disease progresses and the patients develop mobility impairment, breathing problems and corneal clouding.

This report provides the current prevalent population for MPS IV across 28 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Poland, Netherlands, Belgium, 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 IV patients grouped by disease subtype, history of surgical procedures and comorbidities 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 IV include:
• Recurrent upper respiratory tract infections

• Sleep apnoea

• Pulmonary hypertension

• Osteoporosis

• Inguinal hernia

• Hepatomegaly

• Dental caries

• Vision impairment

• Hearing impairment and loss

• Disturbances in urinary function

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 IV market to target the development of future products, pricing strategies and launch plans.

• Further insight into the prevalence of the subdivided types of MPS IV 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.

• Better understanding of the impact of specific co-morbid conditions on the prevalent population of MPS IV patients.

• Identification of MPS IV patient sub-populations that require treatment.

• Better understanding of the specific markets that have the largest number of MPS IV patients.